Welcome to Global Perspectives #1 2025
HAEi’s CEO and Chairman of the Board, Anthony J. Castaldo, and HAEi’s President, Henrik Balle Boysen
Dear HAEi Friends,
We are delighted to welcome you to the first 2025 edition of Global Perspectives – HAEi’s flagship magazine that provides a comprehensive overview of what is happening in the global hereditary angioedema (HAE) community.
Embracing Opportunities across APAC
This edition shines a spotlight on the 2025 HAEi Regional Conference APAC, which brought together about 300 people with HAE, caregivers, and HAE expert physicians from 15 countries across the Asia-Pacific region. Under the theme ‘Embracing Opportunities’, the conference highlights our community’s shared commitment to overcoming barriers and forging stronger bonds within and across borders. With insightful conference sessions and collaboration among people with HAE, caregivers, healthcare professionals, and the pharmaceutical industry, this milestone event has sparked regional inspiration that will resonate globally.We encourage you to explore the full conference coverage in this edition and discover how the collective efforts of our community can transform challenges into real progress by sharpening advocacy efforts, raising awareness, and expanding access to medicines across the APAC region and beyond.
Regional Patient Advocates: Expanding the impact
This year, our Regional Patient Advocates and Advocacy Leads are more active than ever, with bold plans for 2025. With “boots on the ground” in all corners of the globe, Regional Patient Advocates and Advocacy Leads are guiding strategic initiatives to drive awareness, education, community engagement, and access to care. Their efforts include promoting the use of powerful tools like HAEi Advocacy Academy and HAEi Connect, translating key resources such as ‘Understanding HAE’ and ‘Women with HAE’, and empowering local leaders through targeted workshops and policy-focused projects.Importantly, Regional Patient Advocates and Advocacy Leads are inspiring Member Organizations to build bridges between patient groups and decision-makers ensuring HAE remains a priority for governments and healthcare systems. Key events, such as the 2025 HAEi Regional Conference APAC, the upcoming 2025 HAEi Regional Conference EMEA, and the Member Organization Growth & Impact Summit pilot project in South America and Mexico, underscore HAEi’s critical role in uniting our community and fiercely advocating for improving the lives of people with HAE.
Introducing the LEAP Class of 2025
Looking ahead, we’re excited to welcome the LEAP Class of 2025, a dynamic group of 18 young patient leaders who will begin their LEAP journey with a kickoff meeting in April. With guidance from skilled mentors, the LEAP students will participate in a rich curriculum that features training in leadership, communication, community building, along with practical, action-oriented advocacy skills.Together, toward a stronger future
Whether it’s a first-time diagnosis or a life-changing connection made at a conference, every step forward is made possible through the dedication of our community. The stories in this edition of Global Perspectives reflect not just what’s happening today – but what’s possible when we come together, support one another, and embrace opportunity.We are very happy that you are such an important part of the global HAE movement, and wish you happy reading.
Warmest regards,
Anthony J. Castaldo
Chief Executive Officer and Chairman of the Board, HAE International (HAEi)Henrik Balle Boysen
President, HAE International (HAEi)
News from HAEi’s Regional Advocacy team
Michal Rutkowski, Director, Regional Patient Advocate Program
HAEi’s in-region advocates are ready to support transformative HAE community growth in 2025
As HAEi’s Director, Regional Patient Advocate (RPA) Program, my recent focus has been working with our RPAs on their plans for 2025. With more ‘boots on the ground’ than ever before, we set an ambitious course for 2025 to expand awareness, strengthen patient engagement, and improve access to essential resources on a global scale.
Global patient community growth remains central to the RPA Program strategy for 2025. A priority is increasing the adoption of critical educational tools like HAEi Advocacy Academy and HAEi Connect, ensuring they reach Member Countries where awareness and utilization remain low. Digital engagement supports a more connected, informed, and responsive community. The translation of essential educational materials such as Understanding HAE and Women with HAE, alongside the expansion of regional hae day :-) initiatives will further elevate awareness and engagement.
Addressing the ongoing challenge of treatment access remains a key focus of RPAs. The team will deliver targeted awareness projects, virtual local patient workshops, and meetings, designed to equip patient leaders with the skills and resources needed to advocate for change. RPAs will support HAEi Member Countries to increase collaboration between patient groups and government administrations. The objective is clear: to strengthen local networks, drive policy changes, and ensure access to modern HAE treatments.
Regional-level engagement will be instrumental in delivering positive change from HAE advocacy. RPA support and participation in key conferences this year, such as the recently held 2025 HAEi Regional Conference APAC and the upcoming 2025 HAEi Regional Conference EMEA, keep critical issues front and center. The “MOs Growth & Impact Summit” for Patient Leaders from South America and Mexico in May will serve as a key forum for collaboration and knowledge-sharing, reinforcing the community’s commitment to patient empowerment.
By leveraging resources, expanding partnerships, and advocating for improved healthcare policies, our RPAs will work hand in hand with MOs and the wider HAE community to bring lasting and measurable impact. The months ahead will be defined by strategic initiatives that deliver meaningful results for HAE patients worldwide.
I’m excited for the opportunities for significant progress that 2025 presents – stay tuned!
Michal Rutkowski
Director, Regional Patient Advocate ProgramOur team working in the APAC region, Fiona Wardman, Yong Hao Lim, Fanny Schappler, Tony Castaldo, and Henrik Balle Boysen, have been focussed on preparations for and delivery of the highly successful 2025 Regional Conference APAC. The team supported their Member Organizations (MOs) with registration and visa queries, created content for the meeting sessions, and worked with various speakers on their contributions, amongst many other tasks. They are now busy embracing opportunities arising from the conference. You can read all about the results of their efforts in the 2025 Regional Conference APAC write-up in this edition.
News from Sub Sahara Africa
From Regional Patient Advocate Patricia Karani
Since the start of 2025, I have been working with the countries in my region on various different activities. I have supported HAE Comoros and HAE Zimbabwe to make relevant HAE information available in national languages via an HAEi Hosted Website for each country.
In Niger, I have shared information on current modern HAE medication with Dr Hamidou Tahirou, who diagnoses and manages HAE patients in Niger and neighboring countries. We continue discussions on the possibility of accessing these drugs for his patients. Dr Tahirou is looking into the specific protocols and requirements to import modern medications into these countries and what is required.
Additionally, Dr Tahirou continues to train medical personnel in Niger and Burkina Faso on angioedema and HAE to raise awareness and ensure that HAE patients are not missed. To help overcome cultural barriers for people who may have HAE, I started an online group where I can communicate, reassure, and give hope to patients about living with HAE. Dr Tahirou reflects on this approach in talks with his patients.
In Kenya, I liaise with Dr Priya Bowry, Dr Annie Barasa, and clinical officer Grace Polly to support newly diagnosed HAE cases in Kenya. Through my communication with patients, I can support Grace Polly to ensure that patients do not run out of their prophylactic and emergency medications. There is great collaboration across borders, and in addition to HAE awareness and education in Kenya, Dr Priya is training other doctors in Mombasa and Nairobi to correctly diagnose unexplained angioedema cases. Both Dr Priya and Dr Annie are supporting a patient in Mombasa in receiving proper care for HAE.
In my role as RPA Sub Sahara Africa, I receive information on possible HAE cases in various countries. I then have direct discussions with the possible patients and advise them on the correct tests to identify HAE. I explain to the patients what HAE is and link the patient to a local doctor in their country who can continue with investigations. Dr Priya is a huge support and works with the local doctor to establish a treatment plan for the patient once HAE is confirmed.
I’m delighted to report that I’ve had a lot of positive feedback from doctors and patient groups in my region to Global Perspectives being fully online and including a translation feature. Sub Sahara Africa has a diverse range of languages, including Arabic, Portuguese, Swahili, and Amharic, and there is a significant appreciation that the huge information and advocacy inspiration shown in Global Perspectives can be read in these languages.
News from South Eastern Europe
From Regional Patient Advocate Natasa Angjeleska
2025 started with good news for patients in two countries in South Eastern Europe! HAE Albania secured its first access to HAE preventative treatment for two patients (to begin with, we hope), and HAE Kosovo secured patient access to acute HAE treatment for the first time.
As RPA, I supported both countries in their advocacy efforts to access treatment. Specifically for HAE Albania, I assisted in their intensive advocacy activity through the end of 2024. HAEi provided a Letter of support that was sent to the Ministry of Health pointing to the importance of access to modern acute and preventive treatment for HAE patients based on the WAO/EAACI Guideline for the Management of Hereditary Angioedema, as well as the role of MOs in decision making regarding the needs of patients.
I shared data and the presentation from the State of Management in SEE with countries, and having comparable data and information helped different countries in their advocacy efforts with the regulatory and decision-making bodies regarding access to treatment.
I maintained regular communication with pharmaceutical representatives and distributors in different countries, and this was also important in recognizing the need for different therapies in different countries.
The start of a new year offers a natural moment to pause and take stock. I used this moment to remind all MOs in the SEE region about the opportunities to use different resources developed by HAEi.
As a result, HAE Greece started using HAEi Connect, and HAE Turkiye involved other members in maintaining their larger group of patients and caregivers in HAE Connect.
HAE Greece translated the Understanding HAE booklet into Greek. This resource will benefit people in Greece and Cyprus as both countries use the same language.
Other countries have started the translation process with both Understanding HAE and Women with HAE currently being translated into Macedonian, Bulgarian, Serbian, Albanian, Romanian, and Turkish. Both booklets will serve in the awareness and educational activities implemented by MOs.
All MOs are very excited about the upcoming 2025 HAEi Regional Conference EMEA, and I will be encouraging and supporting the registration of patients, caregivers, and physicians from all countries in SEE. The conference provides us with another opportunity to meet in person and discuss new initiatives and plans for action to improve the quality of life of people with HAE and their families. I plan a bigger focus on motivating more youngsters from the region to participate in the specific conference Youngsters Track – watch this space!
News from South Asia, Germany, Austria, Switzerland, and Israel
From Regional Patient Advocate Fanny Schappler
Outside of the wonderful 2025 HAEi Regional Conference APAC, it’s been an active start to the year in countries I support. I’m coordinating many translations of HAEi resources for South Asian countries, and we’re planning in-person patient meetings.
I’m excited to share that as a result of collaboration with the HAEi Team, Afghanistan launched their HAEi Hosted Website, and I supported HAE India with their governance model, which they are now using and recruiting new volunteers.
Following the 2025 HAEi Regional Conference APAC, the MO leaders of the countries I support and I will hold quarterly “South Asian” meetings to continue exchanging best practices and ideas amongst neighboring countries.
I’m also excited to be on the ground, connecting with patients, caregivers, and local doctors when MOs organize face-to-face meetings. Such meetings are instrumental to understanding the reality of the communities we serve and ensuring local MO activities and resources meet the local needs. I’m currently in the planning stages of meetings and visits for Switzerland, India, Austria, and Germany, and I will share more about the meetings in future updates.
News from South America and Mexico
From Regional Patient Advocate Fernanda De Oliveira Martins
Since the start of 2025, I’ve worked with my MOs to update the Regional Medical Advisory Panel (RMAP) contacts in HAEi HUB, as well as checked preferences regarding the use of HAE TrackR and assessed the use of HAEi tools in my region. All these activities aim to improve the use of HAEi tools by MOs and to help them to be more effective in their daily work.
I’ve also been busy planning and preparing for the upcoming HAEi Member Organizations Growth & Impact Summit for Patient Leads from the South America and Mexico region. This HAEi MOs Growth & Impact Summit will take place at the end of May in São Paulo, Brazil. To support fruitful discussions and exchange of ideas, we are working on 2 region surveys; one for MOs and the other for people with HAE or caregivers (if the person with HAE is under 18).
The HAEi MOs Growth & Impact Summit is designed to facilitate RPA and MO close working to identify specific challenges, offer tailored support, and co-create a tailored practical advocacy plan for each region country. Working with MOs to implement their individual plans and through full use of the HAEi toolbox, we can strengthen each organization, ultimately equipping MOs with skills for effective collaboration with pharmaceutical companies and governments on the registration and reimbursement of modern HAE treatments. HAEi and RPAs continually work to support and empower MOs to advocate more effectively for people with HAE and drive meaningful change within their communities.
I’m looking forward to meeting the MO leaders of my region there!
News from Oceania and South Africa
From Regional Patient Advocate Fiona Wardman
I’m delighted we had a strong presence from people with HAE and caregivers in my region at the 2025 HAEi Regional Conference APAC. I look forward to working with my countries on the ideas for activities generated by the meeting.
Outside of the conference, in Australia, New Zealand, and South Africa, part of our ongoing work is to secure funding for projects to continue support for people with HAE and caregivers while strengthening collaboration across borders. The start of any year is a key focus for this activity.
In South Africa, I support the team actively working to secure funding to sustain and enhance the incredible efforts already in place to assist people with HAE and caregivers. The HAE South Africa team’s dedication and completed initiatives have had a significant impact, and we are committed to ensuring they can continue this vital work.Meanwhile, Australia and New Zealand also focus on securing funding for new projects in 2025. I’m working with them on proposals for initiatives that aim to expand support networks, provide essential resources, and enhance patient advocacy efforts in both countries.
Additionally, I support adopting and using HAEi tools and resources for advocacy leaders, people with HAE, and caregivers in the region.
News from Nordics
From Advocacy Lead Jørn Schultz-Boysen
The part of 2025 has brought positive momentum and strong community engagement across the Nordics region.
In Denmark, the year began with a successful patient meeting in Aarhus on 25 March, which included a compelling presentation at Aarhus University on The Meaningful Brain. I had the opportunity to provide some input on the agenda, including ideas for topics. I will continue this support for the two additional patient meetings scheduled before the end of June — one in Odense and one in Copenhagen — which promises continued connection and support among the Danish HAE community.
In Sweden, I am supporting the organization with planning and preparations for their next patient meeting, which will be held in Stockholm on 10 May. The program will feature a physician presentation and other engaging sessions designed to inform and inspire.
A key part of regional advocacy work is sharing experience and advice with organizations to support dialogue with the relevant authorities. I’m delighted to share that Norway is celebrating a significant milestone: a new HAE medication approval—a meaningful development for Norwegian patients. In addition, Norway will proudly have a young representative participating in the LEAP25 program. This is Norway’s first participation in LEAP, and it was great to share information about the program and answer all their questions about it.
Across the three countries, I am also excited about the newly launched SumsUp app! I’ve supported the organizations with questions and testing, and it’s now available as a native app for both iOS and Android. It provides a trusted digital companion for patients navigating daily life with HAE. You can learn more about it on social media, where we’ve actively promoted it to the community.
Sharing information about HAEi’s resources and supporting countries by coordinating translations has been high on my agenda since the start of the year. Finland continues to strengthen their educational resources, having recently translated the Understanding HAE and Women with HAE patient guides into Finnish. I’ve also been supporting Finland with a future transition to use HAEi’s hosted website solution. A youngster from Finland will be a part of LEAP25, and their project will focus on their website, which will be much easier for the youngster to work on directly when HAEi hosts it. Finland’s early April patient meeting included a LEAP presentation from HAEi.
From the start of the year, I have shared information and enthusiasm for hae day :-) with the Member Organizations in my region. As we approach 16 May and hae day :-), MOs across the region are actively participating in the #active4HAE campaign. With walks, bike rides, and creative ways to get moving, the Nordics HAE community is raising awareness and showing their support through action. Seeing so many patients, families, and supporters come together is inspiring — demonstrating strength, resilience, and a united voice for HAE.
Together, we continue to build a stronger and more empowered HAE community in the Nordics!
News from Mediterranean, North Africa, Benelux and British Isles
From Regional Patient Advocate Maria Ferron
2025 has started with a lot of energy! MOs and patient groups are setting their yearly goals and planning and organizing awareness-raising and advocacy activities for the year. I’m on standby to help with any ideas or advice and offer my support or information about HAEi resources if needed.
Here are some of the highlights during this period:
- The patient leads in Belgium & Ireland are working hard to officially register the association in their countries. Through discussions with both organizations, this is a vital step to be able to make progress.
- I have been working with MOs to have the “Women with HAE” booklet translated into Portuguese, Belgian, and Dutch and the “Understanding HAE” booklet translated into Belgian & Dutch. You can now find the translated booklets on the HAEi website under “Resources.”
- Seeing, and in some instances supporting, MOs advocacy activities to celebrate hae day :-) – more to come in the next edition!
- A focus on websites and refreshing information online. Spain has launched their new website (https://angioedema-aedaf.org/) and I’m helping Morocco with the process of updating their web presence.
This period has also been busy with meetings and workshops. The first was on 22 February in Hospital Universitari Vall d’Hebron, Barcelona, Spain. Around 100 patients, caregivers, healthcare professionals & industry representatives participated. I had the opportunity to talk to attendees about the tools, resources, and activities that HAEi offers to the patients and MOs. I also had the chance to talk about the Shared Decision Making tool during the presentation from the Coordinator of Citizen Attention and Participation of the hospital.
The second meeting of 2025 is the 4th National Congress ITACA & A.A.E.E (HAE Italy), from 27 to 29 March in Palermo, Italy. The Congress, organized by the Italian Network for Hereditary and Acquired Angioedema (ITACA) under the patronage of the A.A.E.E., covers key issues related to all forms of angioedema in adults and children and includes the participation of leading national and international experts. HAEi CEO and Chairman of the Board, Anthony J. Castaldo, presented on “HAEi and the global world of angioedema,” and HAEi President Henrik Balle Boysen gave a talk on “HAE and the patient’s journey.”
Last but by no means least, I have been coordinating the first HAEi organized HAE patient workshop in Tunisia. We expect around 70 attendees, and when not working on the logistics, I’m planning a really exciting agenda that will promote disease education among patients and healthcare professionals, including video talks with international speakers and Mohamed Osman, HAEi Advocacy Facilitator MENA, who will give our talks in Arabic.
News from East Asia
From Regional Patient Advocate Yong Hao Lim
The final months of 2024 and the early months of 2025 felt like a brief pause between the 2024 HAEi Global Leadership Workshop and ACARE Global Angioedema Forum 2024 (GLW/GAF) in Copenhagen, and the 2025 HAEi Regional Conference APAC in Manila. Much of my time has been spent on follow-ups from GLW/GAF and preparation for the Regional Conference APAC, an extremely successful event connecting patients, caregivers, young advocates, and medical and pharmaceutical professionals from across the region.
Around my work for the conference, I continued engaging with healthcare professionals to raise awareness about HAE—one of the priority areas identified by MO leaders at the GLW. Highlights were opportunities to share information with physicians at the APAAACI Congress in Kuala Lumpur in mid-December and the Carry Hope Forum, part of Rare Disease Day, in Singapore in February. In Kuala Lumpur, I reconnected with familiar faces while introducing HAE to physicians from Malaysia and Indonesia. In Singapore, I spoke about my experiences as both a patient and an advocate to a diverse audience that included rare disease patients, advocates, policymakers, medical geneticists, genetic counselors, and pharmaceutical representatives.
The prime goal of these speaking engagements is to raise awareness for HAE, but they also provide valuable insights. Speaking with physicians who were learning about HAE for the first time helped me understand their perspectives on the condition, how their healthcare systems can support HAE patients, and their views on second-line treatments. Additionally, I gained insights into relevant topics, such as penetrance and genetic counseling, and identified new opportunities to engage with the medical community.
I have also interacted with HAEi Regional Medical Advisory Panel (RMAP) members, ensuring they stay connected with patient organizations, and I can hear the latest developments in HAE management and patient support in their countries. Through these conversations, discussions are now underway about establishing new ACARE centers in Malaysia, Singapore, and possibly Taiwan, to further enhance patient care.
We are preparing for this year’s hae day :-), and I am strongly encouraging more participation in the #active4HAE challenge. Sharing these experiences raises awareness and fosters a sense of community among HAE patients and supporters. Regular physical activity also contributes to mental well-being—another key area identified by MO leaders at the GLW.
My ongoing work in between the major events is to connect, educate, and advocate—and everyone’s efforts in these areas drive lasting progress.
News from Central Eastern Europe and Middle East
From Regional Patient Advocate Michal Rutkowski
Hello everyone, I hope you have had a good start to 2025!
The past few months were an eventful time filled with activities aimed at supporting Member Organizations (MOs) and patients across the Central Eastern Europe and Middle East regions.
On 9 February, I participated in the HAE Patient Meeting in Abu Dhabi, UAE, facilitated by HAEi. The agenda covered key topics that addressed both medical and everyday challenges faced by the HAE people. Also, the available HAEi resources and future events were discussed. The most impactful part of the agenda was the patient stories, where attendees shared their experiences and lives with HAE. I would like to thank Dr. Marilyn Karam from the Saudi German Hospital in Dubai, the keynote speaker, and Shradha Singhania, the leader of the local HAEi MO, for their contribution to making this meeting possible.
Additionally, over the weekend 8-9 February, I attended the 4th Emirates Allergy and Clinical Immunology Conference in Abu Dhabi. This event offered an excellent opportunity to network with expert physicians from the region. The discussions will help shape future advocacy efforts.
Beyond in-person meetings, I conducted virtual catch-ups with the patient leads of several HAEi Member Countries, including Armenia, Czech Republic, Hungary, Kazakhstan, Latvia, Saudi Arabia, UAE, and Ukraine. These meetings served as an opportunity to discuss the unique challenges each country faces and to offer guidance on current issues and HAEi tools and resources.
In January and February, I engaged with industry partners from CSL Behring, KalVista, and Takeda, through virtual meetings to discuss local and regional issues. These discussions provided insights into upcoming treatments and research initiatives relevant to HAE patients.
A major focus for me has been on HAEi Resources. We keep improving the digital presence of our Member Organizations through HAEi Hosted Websites. In collaboration with HAE Saudi Arabia, we are working on a new, advanced website, which is now in the IT phase. Simultaneously, work has begun on a new website for HAE Qatar. Language accessibility continues to be a priority, ensuring that essential resources are available to more patients. The “Understanding HAE” guide is now available in Persian, and the “Women with HAE” booklet has been translated into Russian. Additionally, we have started translating the Brady Club Activity Book into Arabic for our Middle Eastern MOs.
In Poland, my home country, I assisted in organizing a regional patient meeting that brought together nearly 100 attendees, including patients, caregivers, healthcare professionals, and industry partners. This event, along with two others planned for 2025, celebrates the 20th anniversary of HAE Poland’s official registration and highlights the importance of community engagement in driving reimbursement for modern HAE therapies.
News from Central America and Caribbean
From Regional Patient Advocate Javier Santana
Following the 2024 HAEi Regional Conference Americas held in Panama, the motivation of HAE MOs in the region was high, and we hit the ground running in 2025.
Since the start of the year, patient group leaders have set new goals in their respective countries to raise awareness about HAE. I have supported them with advice on approaching the media, managing meetings with Government officials, and making the most of HAEi tools and resources. As a result, the patient groups plan activities to increase awareness and collaborations with healthcare professional groups, organize education and support sessions for patients and families, plan larger HAE meetings, and use the media to raise HAE awareness.
As part of extensive work from HAE Panama and Dr. Olga Melcina Barrera with the recently elected government and the Ministry of Health, I met with the Office of the Director of Rare Diseases of the Government in February 2025. As a result of everyone’s efforts, the officials confirmed: Panama now has generic icatibant registered to treat attacks, there will be training of more hospital centers about HAE; an official government treatment guide for HAE will be developed, and an official document will be created to help patients avoid problems when seeking HAE services in hospitals.
In the Dominican Republic, last year’s official registration means the group’s leaders have the right to visit healthcare professionals and specialists to discuss HAE. I have supported the group with guidance on how to do this, and since the start of the year, the group’s leaders have contacted physicians in the country to inform them about HAE and to help identify more patients for their group.
The pharmaceutical company CSL Behring recently held a facility event in Puerto Rico for physicians and healthcare providers. I participated in the event as the HAEi RPA for the region. This allowed me to speak with company representatives about their medication plans for HAE in Puerto Rico.
The importance of HAEi’s presence in the region was reinforced early in 2025. A newly diagnosed HAE patient from Trinidad and Tobago contacted me. We discussed HAE and HAEi. I explained how we work, what we do around the world, and how people can get involved. I’m delighted that HAEi has a new member country and patient representative in Trinidad & Tobago. Mr. Zackery Aleong is committed to working with HAEi to improve the quality of life for HAE patients in his country. I’ll support him every step of the way with plans to raise awareness and ensure access to HAE treatments soon.
Welcome to the 2025 HAEi Regional Conference APAC
Proving that every region and country is equally important, HAEi Chief Executive Officer Tony Castaldo and HAEi President Henrik Balle Boysen greeted the around 300 patient, caregiver, and scientific participants from 15 countries across the Asia-Pacific (APAC) region, and encouraged all to apply the theme of the conference by ‘Embracing Opportunities’ to learn, share, and network with their friends and peers.
Henrik reminded the audience that HAEi is motivated by a deep sense of caring and commitment to the HAE community. At the same time, Tony noted that the community is now globally 101 member countries strong, with 17 member countries in APAC alone.
Having first-hand knowledge of the challenges of living with and suffering from HAE, Tony and Henrik made clear that they and everyone in HAEi look positively to tools, solutions, and opportunities.
Tony remarked that the first step towards greater opportunities is understanding the range and magnitude of issues. Before the conference, HAEi conducted our State of Management Survey across the APAC region. From this, Tony highlighted some vital statistics:
- Out of a population of 4.1 billion people in the APAC region, we can expect a population of 137,000 people with HAE.
- Only 3,500 people in the region currently have a confirmed HAE diagnosis: just 2.5% of the potential population. Tony commented that this showed that there is a lot of work to do.
Moving on to knowledge and awareness, Tony indicated that all participating Member Organizations (MOs) felt that general and emergency room physicians lacked a good understanding of HAE. The picture was more positive with doctors specializing in HAE: Three-quarters of MOs highly rated their HAE knowledge regardless of specialty.
Adding to these findings, Tony said that when patients and physicians work together, it creates a tremendously positive situation. More togetherness, he said, leads to more research and, ultimately, more investment from the pharmaceutical companies, which leads to more options to treat and prevent HAE attacks.
95% of identified rare diseases have no treatment whatsoever.
Moving on to the impact of HAE across Asia-Pacific, Tony indicated that 59% of survey respondents felt that HAE had a high impact on daily life, and over half (53%) felt that it significantly impacted patients’ ability to reach their potential.
Tony highlighted that HAE now has 4 modern preventative treatments and 4 on-demand therapies. In addition, there are 3 older medications: androgens, tranexamic acid, and fresh frozen plasma. The survey results indicated 5 countries where the 3 older medications are the only options for patients. For some, importing medicine from elsewhere is the only way to access these older therapies.
Tony told the assembled audience that access to medication is a patchwork quilt across APAC. On a positive note, he showed that one-third of MOs reported access to 1 modern HAE preventative or on-demand therapy.
“Right now, no country in APAC has access to all modern HAE therapies.”
Moving onto advocacy with law makers, data from the survey showed that governments in the region view rare disease and HAE as a very low priority. Tony was adamant that convincing governments to change priorities and provide access to modern HAE therapies is possible. HAEi has a proud track record of successful advocacy action with governments, which is already happening in the Asia-Pacific.
Tony reiterated that availability and access to modern HAE therapies is the number 1 challenge facing the APAC region, but this is a call-to-action. Action is what will make the difference!
Drawing their welcome to a close, Tony and Henrik thanked diamond supporter BioCryst; gold supporters Pharming and Takeda; and silver supporters Astria, CSL Behring, KalVista, and Pharvaris, for their generous support for the conference. Both leaders also took a moment to thank the HAEi organizing team before welcoming Professor Philip Li to the stage for the evening’s keynote address.
Embracing Opportunities for Hereditary Angioedema in APAC … and beyond
Professor Philip Li encapsulated the spirit of the 2025 HAEi Regional Conference APAC in a single sentence: “We don’t only care for our own.” He told his audience that even if it feels impossible, perhaps you are a small clinic or just one patient, you’re never alone. People sitting next to each other right now will be allies today, tomorrow, and for many years to come. This, he said, is the HAE global identity. We care for the entire world’s patients. Those that are doing great must help other countries too.
Before he got to his rallying cry for people to work together, Professor Li highlighted four key areas of activity across the region.
“There are four As: Awareness, Access, Advocacy, and Alliance.”
Asia is a very big and very diverse part of the world, Professor Li said, but there is a common belief that HAE is rare in the East, which he found frustrating. HAE is not rare in the region; it’s just rarely diagnosed. Referencing a review article that included many colleagues from the conference Scientific Committee, he said there aren’t enough specialists, knowledge, advocacy, testing, and medication.
A large part of this is a lack of awareness, he said. Looking at regions like Mainland China and Taiwan, despite lots of genetic similarities, there is a big difference in the epidemiology and how many patients are diagnosed. It’s not, he said, because it’s more common in one region; it’s because the infrastructure and the facilities are different.
How can we improve things? Professor Li told the audience that all the statistics show that countries with patient support groups and guidelines do much better in getting access to therapies.
Professor Li showcased a familiar picture of a patient who was misdiagnosed for many years due to a lack of basic knowledge of HAE. However, advances in HAE in Hong Kong meant the patient was finally able to get the right testing to get a diagnosis. This, he said, was evidence that we know patients are missed, but there is also a chance to embrace opportunity.
Reflecting on the situation in Hong Kong, Professor Li said that as recently as 2018-2019, there were almost no HAE patients, and doctors had no idea what it was. There was no HAE registry, no tests, and no drugs. Patients had nowhere to go. “We’ve managed to come a long way since then,” Professor Li remarked.
One way to embrace opportunities is through education. Professor Li told the patients in the audience that they were more knowledgeable than many doctors in the region. “Work together with interested doctors and educate,” he encouraged them.
Returning to Hong Kong, Professor Li recalled their awareness-raising activities (the first A): “We wrote about HAE in a handbook of medicines for doctors. We wrote about it in local magazines. We taught it in medical school. We went on TV. Patients told their stories. We shared online videos. Whatever it took to teach people and doctors. Our efforts paid off.”
That success was evidenced by the referral pathways, where doctors came forward with an HAE diagnosis in mind. All of this, he told the audience, is down to a partnership between doctors and patients.
As awareness and diagnosis began to ramp up, family screening became a reality in Hong Kong. However, according to Professor Li, there was fear despite the evidence that a diagnosis improves outcomes tremendously. For some people, the fear was due to the needles used to take blood for testing. A new method, called ‘dry blood spot’ testing, removes this obstacle. Now, Professor Li explains, nurses in Hong Kong take taxis to potential HAE patients and get enough blood from a simple pin prick to make a diagnosis.
Expanding on testing, Professor Li reminded people that his second A, access, is not just about medication. Partnering with his colleagues in India has created more opportunities for testing in Asia and has led to clever new options for diagnosing HAE type-2, a rarer form of the disease.
The prevalence in Hong Kong, Professor Li says proudly, is now comparable with the Western world. “You have exactly the same proportion of HAE patients. You just haven’t found them yet,” he told the audience.
Clinical trials also provide a fantastic opportunity to help individual patients and the wider community, Professor Li stressed. “Undertaking clinical trials means patients are the first to try potentially wonderful medication, and we have evidence that they work just as well in Asian populations.”
Professor Li then posed a question: “Are our patient support groups really important?” Answering this, he said that there is scientific evidence that advocacy and having a patient support group and a global network through HAEi improves things. His own research, he said, demonstrated that the establishment of a patient support group leads to more patients diagnosed and greater access to medication.
“If we have a patient support group, we have an identity. We unite our voices. Slowly but surely, you can get treatment.”
As further proof that action leads to positive outcomes, Professor Li indicated that in Hong Kong, there are now prophylactic and on-demand treatments for all of their patients. This is a complete change from 5 or 6 years ago when there were no medications.
Returning to the theme of alliance and working together to embrace opportunities, Professor Li highlighted that by joining apps and sharing data, people are part of something bigger.
“HAE is not just your family. It’s not just your country. It’s across the world.”
Summing up, Professor Li told the audience that education is vital to raise awareness; working across different countries and understanding nuances and barriers can lead to access; stronger patient groups will improve advocacy efforts; and developing regional and international alliances will bring it all together.
“Tell us, and we’ll think of a solution together,” he concluded.
Specialized tracks
After Professor Li’s keynote, the 3 specialized tracks began. The youngsters, led by Nevena Tsutsumanova and Faye Marshall, moved to their specific track to start their program. The healthcare professionals and researchers, led by the Scientific Committee co-chairs Professor Li, Professor Connie Katelaris, and Dr Jovilia Abong, moved to their track. The patients and caregivers remained in the ballroom to kick-start their tailored program.
Continue reading to discover the sessions, speakers, and topics from the Patient and Caregiver Track.
You can read about the Youngsters Track and the Scientific Track using the navigation links.
Enjoy a selection of snapshots from the conference
Use navigation arrows to move between images.
Patient and Caregiver Track
Getting to know each other: Member Organization and Regional Advocacy introductions
Yong Hao Lim and Fanny Schappler, HAEi Regional Patient Advocates, alongside Fiona Wardman, Tony Castaldo, and Henrik Balle Boysen, HAEi Advocacy Leads, took to the stage to talk about HAEi’s Regional Advocacy Program in the Asia-Pacific region.
Yong Hao and Fiona began by reflecting on the 2023 HAEi Regional Conference APAC and the 2024 HAEi Global Leadership Workshop and how valuable it was for Member Organizations (MOs) to meet and share ideas and experiences.
Tony and Henrik explained that in addition to their leadership of HAEi, they support HAE advocacy in Cambodia, Laos, the Philippines, Thailand, and Vietnam. A large part of their work is to strengthen partnerships and support MOs using the activities, tools, and services that HAEi provides. Henrik indicated that their most active locations are the Philippines, Thailand, and Vietnam, with efforts underway to support Cambodia and Laos in finding motivated patients because that is the key to advocacy success.
Yong Hao spoke next as the RPA for East Asia, supporting China, Hong Kong, Indonesia, Japan, Malaysia, Singapore, South Korea, and Taiwan. He offered some highlights from these countries, which included:
- A 2024 annual meeting of over 100 patients in China, the first since COVID
- The success of South Korea in securing increases in available doses of on-demand treatment
- Sites of interest across Japan lit up to raise awareness during hae day :-)
- Regular healthcare professional workshops held across Taiwan
- The proactivity of doctors in Malaysia
- Educational podcasts in Hong Kong
Fanny introduced herself as the RPA for countries in South Asia, including Afghanistan, Bangladesh, India, Nepal, Pakistan, and Sri Lanka. In the six months since joining HAEi, Fanny recalled a recent trip to Bangladesh that led to over 60 doctors and patients learning from each other. Fanny remarked on the incredible resilience of the HAE community and the desire for change.
Fiona spoke next. In addition to her responsibilities in Australia and New Zealand, she also recently traveled to Nepal, where she was awed by the remarkable courage of HAE patients in the country. She also visited Bangladesh to support a physician masterclass before ending her travels in Goa, India, for the ACARE Preceptorship. This is evidence of how interest in HAE amongst healthcare professionals is growing, and she closed by saying, “having motivated healthcare professionals along with active patient advocates is key to collective success.”
Following introductions from the RPAs, each of the MOs introduced themselves in a series of short videos:
Raising Awareness and Advocating for Change
HAEi’s Fiona Wardman offered a simple message to attendees: “Advocacy is for each and every one of us.” Every patient and every caregiver has a job, she said, in helping raise awareness and advocating for the change we need to improve diagnosis and access to medication.
“As a patient, nobody can tell your story better than you.”
People listen every time we talk, Fiona told the audience. It is important, then, that we all raise our voices and not rely on 1 person in a country or a region. Giving an example, Fiona showed that a single social media post can lead to thousands of views, raise awareness, and lead to better recognition and appreciation of the needs of people with HAE.
Although it is important to partner with other rare disease organizations, HAE competes with 7-8,000 other diseases for interest and a share of the spotlight. Only by raising our collective voices can we get our needs across, she said.
If all this sounds complicated, Fiona suggested that by looking at what had gone before and the support available, it is easy to get started. “Look at your neighboring countries and see what they’re doing,” she advised. It may be necessary to adapt an activity or message to suit your country, but it will be simpler than starting from scratch.
Fiona also highlighted the widespread support and tools available through HAEi. The Regional Patient Advocates and Advocacy Leads are there to support anyone in the APAC region looking to get started with advocacy, she reassured. Additionally, tools like HAE TrackR will help you get on top of your HAE, and help build up a body of evidence about treatment and quality of life.
Everyone can take the first step. Fiona said it was simple to start a conversation. Speak to your neighbors. Reach out to others who have been down the path already. Ask pharmaceutical companies for their support. Put your case to doctors, policymakers, and government officials.
Finally, Fiona reiterated that by working together, we can create a future. Advocacy doesn’t mean, she suggested, doing something you can’t do. If we all do what we can, such as sharing a story, writing a social media post, getting involved with our Member Organization, or just supporting others, then that future comes closer.
“Advocacy isn’t a role; it’s a responsibility that we all share.”
HAEi’s Fiona Wardman offered a simple message to attendees: “Advocacy is for each and every one of us.”
Shaping policies
“You can’t just think that someone else will do it,” was Fanny Schappler’s advice to the patients and caregivers assembled in Manila as she gave a practical demonstration of working with governments to shape HAE-friendly policy.
It might sound like a frightening subject, Fanny acknowledged, but it is something everyone can do. The first thing to appreciate is that policy work is a marathon, not a sprint. “You need to pack energy for the long term,” she advised light-heartedly.
Before starting, Fanny gave the following definitions:
- A policy is (1) a set of ideas or (2) a plan of what to do in particular situations that has been officially agreed upon by a group of people, a government, or a political party.
- A policymaker is an individual or group responsible for setting and establishing the various rules and procedures.
“You may wonder what the value of influencing policy is,” Fanny said. The benefit is that better policies can mean improved funding for HAE, increased diagnosis rates, and greater access to treatment options, all of which would ultimately improve the quality of life for patients.
Fanny then provided a step-by-step guide for anyone looking to advance the cause of HAE by working with policy and policymakers:
1. Identify and understand your ecosystem
Here, the job is to understand the healthcare system in your country. How does it work? What are the rules? Is there a rare disease policy in your country? Are HAE medicines available to people right now? A lot of this information can be found simply on the internet. Other advocacy groups might be willing to share their knowledge, too.2. Define your desired state
The task here is to be clear as to what you want the future to look like. Answer: What will success look like? Additionally, be aware that if your long-term goal is big, then having manageable steps on the way will ensure people can see progress along the way.3. Find your allies
Identifying and collaborating with others with similar goals is key to success, Fanny advised. Of course, fellow HAE patients are the first step. Other rare disease advocacy groups, doctors, scientists, and pharmaceutical companies could share common interests. Working together achieves greater impact.4. Gather your evidence
Fanny echoed earlier speakers that evidence is the king. Data is essential to securing policy change, she said. Start by identifying what is and isn’t available. Think about how you can work with healthcare professionals and the scientific community to get the right data that will demonstrate the importance of acting to improve the lives of people with HAE.5. Communicate clearly
Good communication starts with understanding the audience. What are your policymaker audiences interested in hearing about? How can your data be communicated most effectively? Repetition is the key here, Fanny remarked.Overall, Fanny told the audience that “Policy change takes courage, time, strength and perseverance, but by having milestones along the way, the overall task becomes manageable.”
Working with governments: A case study
Fiona Wardman then joined Fanny on stage to talk through a policy activity by HAE Australasia. Fiona gave the audience her favorite analogy for policy work, that of building a bridge. We start with a blueprint; then, we need to build strong foundations before the bridge can get us to where we want to be.
The Australian goal was to secure reimbursement for a modern HAE medication. Fiona provided some background on the Australian system, in that there is a scheme that pays for some medications, along with the National Blood Authority. After this scene-setting, Fiona listed some of the many groups and individuals who partner with HAE Australasia: Clinical Immunology Society Australia, doctors, patients and caregivers, and pharmaceutical companies.
Fiona counseled that an important element is to ensure that everybody involved understands and shares the same goal. Whether it is speaking with the media, doing some sort of awareness campaign, or just liking social media posts, it is important that the message is consistent in the ask for policy change.
Referencing another powerful HAEi tool, Fiona said that the free HAEi Connect database makes it easy to contact members and enlist their support whenever there is a request for data, stories, or information from government agencies.
Ultimately, though, Fiona felt the most important element was time. The success of their work in this policy change was 3 years in the making: “Don’t be discouraged. Repeat the same method. It gets tiring, but we must keep proving the point.”
“Governments have no idea what HAE means to a patient. You’re the expert in your own condition. Talk to them about it.”
Fanny concluded by urging the audience not to sit back but to put plans in place, build a compelling story, and keep pushing. Fiona agreed and encouraged everyone to raise their voice together: “You can whisper, or you can yell. With lots of people, someone is going to hear.”
Addressing stigma and cultural challenges
“You may feel like a zebra standing in a crowd of horses. You may feel people are looking at you differently.” Yong Hao Lim describes how the sensitive subject of stigma and cultural challenges can impact the lives of people with HAE, by making them feel like they stand out amongst others.
Yong Hao told the audience that although he would talk about stigma rationally, it is essential to understand that you mostly feel it as an emotional experience.
He explained how there are different types of stigma. The one we’re most familiar with comes from other people: how people look at us. This is a public or social stigma, where negative attitudes can become discrimination. HAE patients may feel this judgment of others in social situations.
Yong Hao described another type of stigma: internalized or self-stigma, based on the attitudes and beliefs that people with HAE and their caregivers carry about themselves. Research shows that this type of stigma can be the source of mental health issues, Yong Hao said. It shows up as fear and shame in the quality-of-life surveys conducted amongst people with HAE. A recent study showed that people in Japan reported that stigma was high, along with mental health issues such as anxiety and depression.
Yong Hao then described the complicated cultural considerations across the Asia-Pacific region. Yong Hao identified 6 points, focusing predominantly on Asian culture and attitudes.
Loss of face
Aligned with reputation, not just of the individual but of their family and extended family. A condition like HAE may affect an individual’s reputation, dissuading people from seeking help.Traditional beliefs
Very broadly, this is how different cultures view things. The beliefs may manifest in a fatalistic way of looking at a health condition like HAE and prevent people from trying to improve their situation. Other beliefs could suggest HAE is bad luck, bad karma, or even the result of something your ancestors did. All this can lead to a person blaming themselves.Traditional medicine
For some people, there is less reliance on science-based medicine and more belief in the value of traditional medicine, such as eating certain foods or using herbal preparations. Whatever the form, this can prevent people from accessing objectively proven effective treatment such as modern HAE medicines.Paternalistic healthcare
Paternalism in healthcare describes a relationship where all power resides with healthcare professionals who make all the decisions for a patient. “Trusting in your healthcare professionals is vital,” said Yong Hao, “but there should be shared decision-making, in which the patient and the doctor have a meaningful conversation before making an informed decision.”Collectivist values and interdependence
Yong Hao spoke about the idea of burden, as someone with a diagnosis can see themselves as imposing a burden on others, such as their family. In this view, everyone sees themselves as part of a larger social unit and decides if their diagnosis will negatively impact their friends and family.Marital expectations
Finally, Yong Hao described cultural considerations related to marriage and children. There can be issues if people feel that a genetic condition like HAE will be passed onto children, and the effect that might have on the desire for a wider family line.Im-Kyung Lee’s experience and advice
Im-Kyung Lee, from HAE South Korea, joined Yong Hao on stage to discuss her experience of cultural stigma and challenges.
For Im-Kyung Lee, South Korean society means some people may perceive HAE as a burden or view it negatively. As HAE is a genetic disease, there are concerns about people getting married and having children. Some people may wonder about the impact on future generations. All this means she feels burdened when explaining her condition, and resented HAE as it constantly meant explaining she had not brought the illness on herself. All this cultural stigma made her feel hesitant when meeting new people.
All this changed when she joined HAE Korea and learned more about the disease. She felt more confident in explaining that the disease is not something to be looked down upon: people with the condition can live their lives without constraints as there are effective treatments. Over time, people accepted her and her illness, treating her with empathy and understanding. Her current boyfriend is different from others who questioned her suitability for marriage. He tried to understand the disease and learned how to inject her in case of an emergency.
She strongly advised everyone present to join their country’s patient support group and connect with people with similar experiences. “This will be a great source of strength and support,” she counseled.
Finally, she encouraged everyone to have a positive mindset, as it is not helpful to be frustrated or negative. Continuing to learn and understand means you will have more confidence in your own health. “HAE is simply part of our lives; have faith you can live a normal life like everyone else,” Im-Kyung Lee concluded.
How to address stigma and cultural considerations
Yong Hao described ways in which people can address the issues that stigma can cause, and how cultural notions may prevent people from pursuing the best outcomes for their HAE.
He started by suggesting that it is important to collaborate with health experts, whether traditional or modern medicine, religious experts, and other cultural leaders. Working with these individuals will communicate a sensitive message about the disease and its appropriate treatment, which can be powerful in the relevant communities.
In terms of stigma, Yong Hao reiterated the importance of knowledge. Joining a patient group will provide peer and community support and opportunities to educate yourself. All these activities can reduce self-blame and stigma and empower people to seek the treatment they need to improve their lives.
Im-Kyung Lee from HAE South Korea and Yong Hao Lim from HAEi talked about how stigma and cultural challenges can impact the lives of people with HAE.
Ganesh’s story
You might remember Ganesh Dahal from the last 2024 edition of Global Perspectives when he took us through the development of HAE Nepal.
From the stage, Ganesh told his story to the assembled patients and caregivers in Manila.
He told the audience that he was born in Kathmandu in 1995, and not long after his birth, the family lost his father to undiagnosed HAE. He said it was a terrible blow and the turning point of his life. Ganesh had his first attack at age 12 and only received an HAE diagnosis 10 years later. He paid tribute to his resilient and wonderful mother and his beautiful wife, Alisha, who were in the audience listening.
Despite the challenges he experienced with HAE, he became the first person in Nepal licensed to practice accountancy in the US. He shares this as he believes that it is possible to empower people and, as he says, swell with purpose.
He concluded by telling the audience that he helped start HAE Nepal as an opportunity to serve people and improve advocacy in his country. He also expressed his gratitude to Fanny and Fiona from HAEi and physicians Dr Dharmagat and Dr Sudeep from Nepal for their support in creating HAE Nepal.
You can read our in-depth feature on HAE in Nepal, from both the patient and physician perspective, here.
BioCryst’s promise
Charlie Gayer, Chief Commercial Officer for BioCryst, took to the stage, as he had done in Copenhagen for the 2024 HAEi Global Leadership Workshop. But Charlie wasn’t alone. His colleague Jinky Ang Rosselli, Chief Data Insights Officer, was also there, and for her, a conference in the Philippines was something of a homecoming.
Jinky told the audience that she was born in part of Manila and moved with her family to the US at a young age. She recounted the importance of family and gratitude in Filippino culture, and how working at BioCryst and hearing every day from the HAE community feels like an incredible gift.
Jinky said that there are several healthcare professionals in her family, so she understands the need to serve a community. She and her team do this in HAE by listening and learning from people with the disease to ensure the story is as complete as possible.
“We try to humanize the data and understand the communities we love.”
Jinky said that part of listening is understanding what the HAE community wants. And what BioCryst has learned is that the community wants therapies that work, especially as HAE is so unpredictable. Charlie and Jinky accepted that, in this part of the world, access to therapy is limited, and they are working hard on that.
Charlie then promised the audience: “We are going to make our drug accessible regardless of patients and the healthcare system’s ability to pay because we think it’s really important to have access. We don’t know how long that will take, and we thank you for everything you’ve done for us.”
BioCryst is a Diamond supporter of the 2025 HAEi Regional Conference APAC.
An HAE travelogue – Evelien’s story
The 2025 HAEi Regional Conference APAC audience welcomed Evelien to the stage. Evelien is an HAE patient in Australia who shared a cautionary tale about traveling with the condition.
Evelien told the audience that her family is from the Netherlands, but she has been living in Australia for 25 years. For her father’s 80th birthday a couple of years ago, the whole family decided to go on the trip of a lifetime to Tanzania in Africa.
“It was wonderful, amazing!” she remembered. “We went on safari and saw the lions and elephants and giraffes. We had a very good time.”
Unfortunately, Evelien remembers the idyllic holiday for another reason. After a snorkeling trip, she started to feel a bit of swelling. She thought nothing of it, but the next day, her eyes became swollen, and then, most worrying, she felt pressure on her throat. Concerned, she rang Fiona Wardman of HAE Australasia (and HAEi).
There was quickly a realization that there was no medical option. South Africa’s hospitals were a 6-hour flight away. “I was in a lot of trouble,” she remembers.
Luckily, her sister and fellow HAE patient had on-demand treatment with her ‘just in case.’ Evelien got the intravenous treatment and the next day felt much better physically. But, Evelien deeply regretted how she put herself (and eventually her sister) at risk.
Evelien made it clear that she knows what she needs to do. She’s an active member of her Member Organization, is proactive with her medical care, and has been on prophylactic treatment for 30 years. In that different country, she said, you never know what position you’re going to be in.
“I’ve learned my lesson 100%. I travel with prophylactic and on-demand medication. I have it with me now. I don’t want to put myself in that situation again.”
Evelien was lucky that her sister and fellow HAE patient had on-demand treatment with her ‘just in case.’
HAE Travel and Relocation Guide
With Yong Hao Lim running a little late from giving a talk in the Scientific Track, Fiona Wardman briefly stepped in and introduced the next session, which looked in greater detail at the key things to know before traveling or relocating to a new country.
“Make sure you’re prepared. Speak to your physician. Let them know. You can’t talk to too many people about this.”
You can also simplify preparation by using essential apps like HAEi Companion. This app gives you information about the countries you want to visit, with emergency cards in local languages, to advise hospitals about your HAE and what to do.
Yong Hao had joined the session and took the microphone, recognizing that travel can still be impossible for people with HAE. A patient from Malaysia was unable to participate in this Regional Conference due to a lack of treatment for her unpredictable attacks. However, the situation is changing.
Fiona and Yong Hao agreed that research can connect you with local resources, as can contacting your HAEi Regional Patient Advocate. “We can help direct you to the right person,” was Yong Hao’s message.
Returning to HAEi Companion, Yong Hao showed how it lists all the places in a country with knowledgeable HAE physicians, but their clinic or department might not be the right place to go in an emergency. You should prepare and have all your documents (letter from doctor, insurance certificates, etc) in hand and to check what your insurance covers before you depart.
Other tips from Yong Hao on managing HAE when you’re away from home:
- Plan for the worst rather than just hope for the best.
- Educate as many people and agencies as possible. If you’re relocating and have children with HAE, the school may need a lot of education.
- Get the medication you might need in advance, and find out if there are resources to appropriately store treatments (such as constant refrigeration).
- Get your documentation in order before you go. Make sure you have letters explaining your condition and treatment. These letters could be helpful for customs, not just healthcare professionals.
- Use all the support HAEi supplies. Everything is readily available through the website.
You can find, read more about, and download the HAE Travel & Relocation Guide on HAEi’s website.
Patients and caregivers panel discussion
A group of patients and caregivers from across the Asia-Pacific region assembled on stage to give their views and experiences of life with HAE. Our 13 panelists represented 12 countries from across the region:
Leanne from Australia
Ovi from Bangladesh
Mdm Zhang and her husband, Mr Qi, from China
Shashwat from India
Rudy from Indonesia
Makiko from Japan
Alisha from Nepal
Olivia from New Zealand
Krizzia from the Philippines
Yong Hao from Singapore
Soojin from South Korea
Suchi from ThailandThe session covered challenges, changes, and hopes for the future. The first question was: What has been the biggest challenge in managing your HAE, and how did you overcome it?
Rudy from Indonesia spoke first. He said that the availability of medicine is the biggest challenge in his country, as there’s no HAE therapy. He depends on medicine from other countries, such as Singapore, Hong Kong, and Thailand.
Shashwat advised that the biggest challenge is stress, as it is the biggest trigger for his HAE attacks. He had problems as a student as the stress of exams would bring on attacks. Thankfully, he said, his professors and teachers were aware of his disease and knew what to do and what medicine to give.
Alisha, the wife of HAE patient Ganesh (who spoke earlier in the day), was asked about challenging moments as a caregiver. She said the lack of support in Nepal was a real problem and that Ganesh’s condition is managed according to symptoms rather than prophylaxis.
Krizzia was asked what she would want policymakers to understand about life with HAE. She believed that healthcare providers need to know that HAE affects daily life beyond physical symptoms. The uncertainty of attacks can impact mental health, work, and relationships. She would also ask for more awareness and education among healthcare providers, as this would make a big difference in early diagnosis and treatment.
Olivia was asked about her experience as both an HAE patient and the mother of a child with HAE. She felt that because she had personal experience with the condition, she was always trying to ease the way for her children. She does this by leading by example. Her son was with her at the conference to see other people with the condition living and thriving. Reinforcing the message about traveling with HAE, Olivia stressed the importance of organization and planning.
Makiko was asked how she manages to parent a child with HAE. She says that educating teachers is essential for her. She arranges orientation sessions with them to discuss managing attacks and treatment. But her instructions to them is that they call her first, and then they have nothing to worry about because she will act.
Soojin was asked about how she shares her HAE with other people. Responding in her native Korean, Wordly provided a translation to those present: Korean society can make it challenging to be open about a genetic disease; it can be stigmatizing because people may have negative views of genetic illnesses. In the past, she found it hard to tell colleagues or employers as it might have caused work and promotion issues. Now, she tries to spread the word about the disease as much as possible so that many more people understand the disease.
Rudy felt that people were interested whenever he started to tell his story. He tells his story to all sorts of people to inspire them. If there is hope, he believes, anything can happen. Rare disease stories are different and can inspire people, Rudy concluded.
Yong Hao took a break from his HAEi responsibilities to give his perspective from Singapore. Switching to Mandarin, the Wordly translation tells us that Yong Hao initially thought his symptoms might result from an unhealthy lifestyle. However, after his diagnosis, he knew that it had nothing to do with his behavior. Yong Hao stopped blaming himself. The diagnosis also made it easier to share his condition with other people, so he tells new students that he teaches. He said he proactively shares HAE information with everyone rather than waiting for them to ask.
Turning to Mdm Zhang, an HAE patient from China, the question was about the most valuable support she receives. Again, in translation from Mandarin, Mdm Zhang responded that the HAEi conference in Denmark was the first time she didn’t feel alone. Before that, she wasn’t aware of a second person with HAE in China. Now she knows many people around the world have this ‘strange disease.’ And these people were not like patients; they were very happy. Everyone was smiling. She gained strength from this. Now, there is an HAE Organization in China with many members who help and support each other, from the big international family to the small family in China.
Continuing the theme of community, Leanne from Australia spoke about her experiences as a caregiver to her son Levi. She remembers that around his diagnosis, there was little information and advice, but HAE Australia connected her with other mums, which was a real blessing. She recalls that they had so much more advice and insight than any doctor. It gave her peace to know that her son would be OK.
Suchi from Thailand shared this view. She said the patient support community really helped her as a caregiver, sharing information on new treatments and how to deal with challenges, such as convincing policymakers to pay for medication.
Mr Qi was asked to share his experience as a caregiver to his wife (Mdm Zhang). He spoke about the importance of being a good listener and being there for her when she needed to travel to meetings or conferences. In the past, this was harder because of his work, but now that he is retired, it is easier. While working, he remembers difficulties, but ultimately, she was more important to him.
When asked about the advice she would give other caregivers, Alisha said the first thing was to know about the disease. Go to people who understand the condition and learn from them. Then, she felt it was essential to have patience and to be strong as a family.
The topic of conversation moved to barriers to accessing treatment. Even in Australia, not everyone gets access, Fiona remarked. In Singapore, Yong Hao said, the vast majority are still struggling to get the proper treatment or enough treatment, as the disease can be very different, and one medicine does not work for everyone.
Suchi shared her tips on navigating medical systems and advocating for better care. She felt that you must be an educator. Learn about the disease because if you don’t, policymakers will never understand. Working with as many partners as possible is also important, she stressed. You cannot do it alone. You need friends around you.
Shashwat was asked to comment on a time when he advocated for himself in a medical setting. Highlighting that medication is not available everywhere in India, and that there is currently no self-administration, he felt that advocating for yourself meant finding a good doctor with expertise in HAE. Ask them questions about medication upfront. A good doctor can speak for you when you need medicine, perhaps in a different hospital.
Olivia was asked the same question. She felt that the most important thing she’d learned was not to be complacent. You never need permission to advocate for yourself or to question your doctor. She recounted stepping in to advocate for an elderly relative who required extensive dental surgery. Doing so felt awkward, she said, but she worried more about the consequences of saying nothing. Ultimately, her speaking up led to a better outcome.
Krizzia was asked to comment on the impact of a patient organization on her HAE journey. She told the audience they are working on a patient group in the Philippines to build greater awareness, which is the most important job right now.
The HAEi team commended the ongoing work in the Philippines, reminding everyone that advocacy is an ongoing job. It’s never over, they said. Whenever you think it’s over, there will be a new diagnosis or treatment.
Yong Hao felt that being part of a patient group in Singapore meant he was listened to more. The numbers may be small, but when people came together, it created opportunities to join conversations, such as with policymakers.
The changing role of caregivers was discussed next. Makiko, in translation from Japanese, said that over time, with improvements in the treatment environment in Japan and the availability of preventative treatment, her daughter is free from attacks. This has changed how active she has to be as a caregiver.
Likewise, for Leanne, her son Levi is also on prophylactic treatment and has been attack-free for 2 years. At diagnosis, the outlook was grim, but now he can enjoy things like getting excited for Christmas and birthdays, just like normal kids do. I’m grateful for how far we’ve come, she said.
Asked about how Levi’s HAE affected her other children, Leanne said that it was very tricky. The other children would have to spend time with their grandparents whenever there was an attack. Levi’s siblings would see ambulances at school and assume they were for Levi.
Suchi was asked about how she supported patients. She recounted how important it is to speak up and to be proactive and strong in advocating for others. Suchi recalled a situation with a fellow caregiver struggling to get the appropriate care for her son. Suchi called doctors and hospitals to get the proper treatment for the patient.
Mr Qi was asked about how he supported his wife emotionally. He said it is important to understand the situation and try to do more, such as talking to the doctors to reiterate her challenges. He also felt it was important to give her time.
Ovi was asked to look to the future. In Bangladesh, Ovi said, the situation is changing gradually. The hope for the future is that society will better support the underprivileged so that they can have a diagnosis and access to medicine.
For Yong Hao, the future will be positive for his daughter. If she is diagnosed with HAE, she is very likely to have a normal life. Soojin shared this positivity for the future, both for herself and her son.
That message of positivity ended the panel discussion. The HAEi team thanked everyone who participated, reiterating how modern HAE treatment can improve lives and how much they hope this will soon be available to every patient across all the countries present.
13 patients and caregivers in the panel represented 12 countries from across the APAC region and gave their views and experiences of life with HAE.
Final sessions of the conference
On Saturday afternoon, the 3 tracks came together for the final sessions of the conference: Talks and Q&A with HAE experts from the 2025 HAEi Regional Conference APAC Scientific Committee.
10 things HAE patients need to know
Dr Hilary Longhurst, an HAE specialist in Auckland, New Zealand, stepped on stage to give the first of 4 much-anticipated healthcare professional talks.
She introduced her talk as the ’10 things HAE patients need to know’, compiled from the questions frequently asked by HAE patients.
The first questions she addressed were: Why me? What causes HAE attacks? The short answer, she said, was that most people with HAE have a faulty gene called SERPING1. This faulty gene means people don’t have sufficient C1 inhibitor protein. Dr Longhurst told everyone that blood vessels are designed to be leaky at the right time and allow oxygen and nutrients to enter our tissues and cells. In people with HAE, the body produces too much of a substance called bradykinin because the C1 inhibitor protein is absent. The bradykinin tells the blood vessels to leak, which in turn leads to angioedema, and that’s what causes HAE.
The following questions were: How can I recognize the early signs of an HAE attack? What should I do immediately?
Dr Longhurst asked the audience if they knew when an attack was coming, and quite a few people said they did. Dr Longhurst noted that this is something that happens, and seemingly more so in younger people. The things that happen include ring-like rashes or patches on the skin, feeling irritable, numbness at the site of a forthcoming attack, and sometimes an overwhelming sense of fatigue. “That fatigue is perhaps not surprising,” she said, “your body is putting oxygen and energy into parts where it shouldn’t be.”
All of these and many others are examples of prodromal or warning symptoms.
Attack warning signs are individual to each person, Dr Longhurst suggested, and the biggest mistake is when patients leave it too late to treat. “Perhaps they felt some stomach discomfort and tried to sleep it off,” she said. The problem can be two-fold: (1) It can take a lot longer for everything to settle down after treatment, and (2) there can be a higher chance of the attack coming back. Dr Longhurst advised the audience that, where possible, “Treat early. You don’t know how bad that attack is going to be.”
“Know in your head what needs to happen if you have an attack. Get your paperwork together. If you have it, find your attack medication. Plan your day, because there may be disruption.”
Next up was the question: When should I seek emergency medical help?
The first thing to think about, Dr Longhurst said, was pain. If you’ve got such incredible pain you can’t function, just go to the hospital. Dial the emergency number for your hospital.
The next area of immediate concern is throat attacks. These are very, very dangerous, Dr Longhurst said. Although rare, you need to know what to do to protect yourself. If you feel something not quite right in your throat, she said, treat any swelling inside the mouth immediately.
“If you are having a throat attack, get to the hospital. Don’t finish what you’re doing. Don’t finish your shopping trip. You need to go straight to the hospital.”
When you get to the hospital, this is where a letter from your doctor or the HAEi emergency cards come in. You might not be able to speak. These cards, available in many languages, can be easily shown to doctors.
Dr Longhurst tackled triggers: What common triggers should I avoid to prevent HAE attacks? She started by indicating that medicines called ACE inhibitors will almost always lead to more HAE attacks. Although ACE inhibitors are commonly used for blood pressure treatment, doctors can prescribe other effective drugs. The second is medicines containing the female hormone estrogen, such as birth control pills, hormone replacement treatment, and some cancer treatments. There will be alternatives, except perhaps in rare cancer cases where your doctor will help you manage the risks.
Beyond these medicines, for patients on preventative treatment, Dr Longhurst didn’t think patients should avoid potential triggers. “Proper prophylaxis means you can do normal things,” she said.
However, she acknowledged that you can’t always avoid certain triggers. Stress and menstruation (with its associated hormonal fluctuations) were 2 examples. Dr Longhurst’s advice was if in doubt, take your medication. She also used this opportunity to answer the question: What should I do if I run out of medicine or can’t access medical help easily? Plan ahead, was her advice. The guidelines recommend that people have at least 2 treatments. “Keep that treatment with you, especially when you don’t think you’ll need it,” said Dr Longhurst, “as that will be exactly the time an attack might start.”
‘Why is it important to get my family tested for HAE?’ was the next question Dr Longhurst faced. The simple answer is that if you have a diagnosis, your chance of dying from HAE -even if you don’t have treatment- is very, very much reduced. The more you know about HAE and what you can do to manage it, the less likely you are to die of it.
Even in a country with only basic treatments available, this can make a huge difference: using these treatments can reduce abdominal attacks and help you get the exam results you need or keep a job – all the things that make life worth living.
Dr Longhurst moved onto the question: Who can I turn to for help when I feel isolated or overwhelmed? Referencing the song forever linked with Liverpool Football Club, ‘You’ll Never Walk Alone,’ she told the audience that they, too, are not alone. “If you feel bad, contact HAEi, your family and friends, or your doctor. Email me,” she said, “I respond if someone emails for help.”
The last question was: Will my life ever feel normal with HAE? Dr Longhurst commented wryly that perhaps normality was overrated. Even before modern treatments, she had patients who became doctors, head teachers, and academics. People found a way to live their lives. She recalled a patient who sailed the South Pacific with her 2 teenage kids.
“You can still do a lot of things,” she said. “Life may not be normal, but life can be really full and fulfilling.”
Looking to the future, Dr Longhurst said that life will be normal or much closer to it: “We are working together to ask people to provide all HAE patients with what they need to restore normal health.”
“You can still do a lot of things,” Dr Hilary Longhurst said. “Life may not be normal, but life can be really full and fulfilling.”
Finding the index patient in Jammu, India
Dr Ankur Jindal from Chandigarh in India was the next HAE expert to speak.
In his presentation, he talked about families with HAE in the northernmost state of India, Jammu.
Dr Ankur suggested that HAE is a disease that is grossly underdiagnosed in India and across the Asia-Pacific region. To date, experts have been able to diagnose about 700 patients in India, but they believe there are more than 30,000 patients in the country still to be found. “We have a lot of work to do,” said Dr Ankur.
He spoke to some of the challenges around HAE in India and the Asia-Pacific region. These include:
- Low awareness
- Limited diagnostic facilities
- Availability and access to treatment
Moving on to the families in Jammu, he repeated the positive diagnosis message Dr Longhurst gave. In the patients shown to have died in these families, the only difference was that the deaths were patients who had not been diagnosed with HAE. “If you have a diagnosis of HAE, your risk of dying is very, very lessened,” said Dr Ankur.
The index (or first) case of Dr Ankur’s talk was a 21-year-old woman from Jammu, referred to him during her pregnancy. She, her husband, and her family were worried about symptoms of recurrent angioedema and whether that would be transmitted to the unborn child. She was diagnosed with HAE, but their only concern was to prevent the next generation from being born with the disease. After genetic testing, they chose to terminate the pregnancy. In the second pregnancy, after genetic testing, the child was found not to have HAE.
This young woman was the first. She remarked to Dr Ankur that these symptoms -and so HAE- are very common in their area. People accepted it as part of their lifestyle, but no one was diagnosed or treated.
“How can a rare disease be so common in one particular small area?” thought Dr Ankur, a question he kept returning to. The first concern was supporting these patients, who were reluctant to come to his hospital. Instead, he and a team made the first of 3 field visits to the area to diagnose cases. They diagnosed many patients and found an unusually high proportion of females, something that they still don’t understand. All of them carried the same mutation of the gene.
Despite offering advice on treatment, when they returned, none of the patients were taking treatment. They couldn’t afford medication. They did not understand the severity of the disease, seeing it as just part of their lives. To address this issue, Dr Ankur and his team worked with local doctors in the region to teach them how to manage patients. This helped, but the motivation fell in the following months. On their third trip, they contacted families directly again and were able to support them with medication, including C1-inhibitor for some patients. Dr Ankur told the audience that the area has challenging terrain, bad roads, and high mountains. All of this makes it difficult to get to the hospital.
“There are people who think about you. You might have lost hope. We haven’t.”
Through the help and support of HAE India, Fanny Schappler, and Fiona Wardman from HAEi, Dr Ankur and his team are committed to supporting this particular region. Dr Ankur and his colleagues will persevere to break social stigma and provide more treatment options to give people hope.
“There are people who think about you. You might have lost hope. We haven’t”, Dr Ankur said.
HAE medications – current and new medicines
The penultimate of the 4 expert speakers on Saturday afternoon was Professor Philip Li, whom the audience first heard in Friday evening’s keynote. He introduced this talk as focused on medications. He acknowledged a very varied audience, with both experienced experts and people new to the disease, and the presentation would be broad.
Referencing Dr Longhurst’s presentation, Professor Li showed that many existing medications resolve leaky blood vessels caused by too much bradykinin. He told the audience, “The problem is that it’s tough to stop once it starts. It is why doctors tell patients they need to be treated early.” Professor Li explained that all the medications currently in development are trying to stop the whole process.
Medications for HAE come in 2 categories, Professor Li indicated. These are on-demand, given when a swelling occurs. The other is called preventative.
Before deciding what you should get, Professor Li counseled that you need to know what you shouldn’t. Many people with swelling get antihistamines, steroids, or adrenaline. These will not help at all with your HAE attack. He said that if you go to the emergency department, be alert if they don’t believe or understand your HAE.
Referring to the latest available guidelines from 2021, Professor Li indicated that the most straightforward way to treat is by replacing the C1-inhibitor in people with HAE, where they either don’t have enough C1 or the C1 they have doesn’t work correctly. C1-inhibitor is usually given as an infusion into a blood vessel. The good thing about C1-inhibitor replacement, according to Professor Li, is you are ‘nailing the problem.’ All patients can use C1-inhibitor, even children, pregnant women, and breastfeeding mothers.
Probably the most well-known HAE injection is the next treatment Professor Li described, icatibant. “It’s very good,” he said, “you can do it at home without a doctor, but pregnant women or those breastfeeding can’t use it.”
He briefly referenced a medicine limited to the US as an example of limited options for on-demand treatment.
Professor Li then moved on to the various ways you can use medicines to prevent swelling attacks from happening at all. For short-term prophylaxis, when an attack might be anticipated (such as after dental work), an infusion of C1-inhibitor can prevent an HAE attack.
Looking at long-term prophylaxis, preventing attacks over a long period, this field is growing, according to Professor Li. You can have regular infusions of C1-inhibitor or a special form of C1-inhibitor that absorbs slowly, giving more extended protection than twice-a-week infusions. A subcutaneous injection or a daily oral tablet is now used to prevent HAE attacks.
Professor Li moved the focus to look at how HAE treatment is changing. He acknowledged that some countries currently have nothing and stressed the importance of advocating for HAE-specific treatment.
He started by looking at his native Hong Kong. Here, he said, the approach was to secure treatment that prevented death. He wanted to save lives and relieve attacks. Now, he said, the focus is on gaining control. ‘Can I live a normal life?’ is now the question. Perceptions have changed. Patients and clinicians are considering the right medication for each patient and the treatment that best fits their lives. “For example, a patient might want the ability to treat attacks at home, or an injection might not be suitable,” he suggested.
He indicated that future medication could be summarized around 4 themes:
On-demand pill in a pocket
Sebetralstat and deucrictibant are the options being tested for this.Prophylaxis with fewer injections
Garadacimab (every month), donidalorsen (every 2 months), and navenibart (every 3-6 months) are in clinical trials here.Prophylaxis without injections
Deucrictibant is also being tested as a daily tablet preventative.No more medication at all
Gene therapies that modify the faulty gene in people with HAE are being tested. Professor Li said it is very promising, especially for those who have tried multiple lines of therapy but are still having very severe attacks; however, it’s not going to be readily available everywhere.Wrapping up his presentation, Professor Li admitted to being angry when they finally got C1-inhibitor therapy in Hong Kong. He could see in other countries that they were talking about the normalization of life, which should be everyone’s aspiration. He said that this is why it’s important to know what’s out there so you can see where we should all be aiming.
”It’s important that we all know what medications are out there and what’s coming, so we can see where we should all aim.”
Professor Li encouraged everyone to look at the current trials and join one if they could – to benefit themselves and develop future medication. He also asked the clinical experts to work together on a realistic consensus and advocate for the region, before closing with an invitation for the conference to come to Hong Kong.
”It’s important that we all know what medications are out there and what’s coming, so we can see where we should all aim", Professor Li said.
HAE in women, pregnancy, and children
“With children, we have to set them up for success rather than a life of anxiety and depression and worry about having a genetic disorder.”
Professor Connie Katelaris summed up her view on how to support children with HAE in a single sentence. Her presentation, the final one from our experts, took in the situation of mothers and pregnant women with HAE, too.
Children with HAE
Starting with children, she told the audience that between half and three-quarters of children with HAE have their first HAE attack by the age of 12, and 90% will have had one by the age of 20. This means, she said, that this is a disease that manifests in childhood, and the way in which those early years are managed is really important for the rest of the child’s life.
The earlier the disease starts can be linked to more severe HAE, she said, but females also tend to show symptoms sooner in life.
Diagnosis in children can be delayed, she said, because there is a low awareness of the condition. In the case of abdominal symptoms, these can be attributed to much more common causes of recurrent abdominal pain in children, like appendicitis.
Professor Katelaris stressed that children often have prodromal symptoms, such as a non-itchy raised rash. These sometimes occur long before a first swelling attack. The actual attacks themselves are exactly the same types as in adults. The most common locations are the abdomen or the hands and feet. Laryngeal attacks are thankfully rare, she told the audience, but they do occur and are concerning because of the small size of a child’s windpipe. All throat or laryngeal attacks are an immediate emergency, she said, as the airway can be compromised much more quickly in a child.
Moving to diagnosis, Professor Katelaris outlined the importance of medical and family history. Biochemical marker tests are measured, but in her view, genetic tests aren’t usually necessary except for the very rare form, HAE with normal C1-inhibitor. For a typical presentation, family history, and biochemical markers, there is no need for genetic testing of a child.
After diagnosis, she recommends 2-3 visits with the doctor to go through everything, which includes:
- Pointers on recognizing attacks
- Discussing prodromal symptoms and trigger factors
- Treatment and management options (where this luxury exists), with input from parents, caregivers, and the patient if they are old enough
- Getting a written management plan so everyone knows what is agreed. This can also be helpful for doctors in the emergency department, Professor Katelaris noted.
- The national HAE organization and the importance of joining
- Equipping the child with the skills and knowledge to understand the condition, explain it to others, and manage any anxiety
Touching briefly on treatment, Professor Katelaris noted that not everyone in the audience had access to HAE-specific therapies and hoped that would change in the coming years. Whether you have or don’t have effective treatment, she said, there is a great deal we can do for children. A key part is to help them make sensible choices. Using sports as an example, she mentioned a patient who was a very aggressive player in a contact sport, which meant he ended every game with a swelling, making it not a great choice. Talking about something in which you can succeed and not be impaired by HAE is really important.
Hearing and addressing the worries every child with HAE had was also important to Professor Katelaris. She asks all young patients to tell her what they’re worried about; sometimes, it’s sleeping over at a friend’s house for fear of an attack, or being embarrassed for their friends to see their hand or foot swollen. Professor Katelaris suggested that managing anxiety is very important and should be part of overall healthy living (with or without HAE). This, for her, involved a good diet, good sleep, and good exercise.
Help yourself to get good sleep; have a regular bedtime, with no phones or devices before bed. Perhaps reading a book and learn how to relax from the day’s activities.
Another good practice is to look at the family dynamics, Professor Katelaris counseled. If parents are overly anxious or worried, especially if they also have HAE, a child will learn to feel the same way. A more balanced attitude will help the child see a better way to approach a lifelong disease with less anxiety.
Mentioning triggers, Professor Katelaris highlighted that climbing trees and having falls may lead to attacks, but it’s a normal part of childhood. Children also get lots of common infections. This can’t be stopped, but making sure children with HAE get all the immunizations that are available will really help. The number 1 trigger was, she said, stress. We all have stress, she said, but increased disease activity leads to anxiety, which in turn can lead to increased risk of another attack. If you have a child with HAE who is more nervous or anxious, they might need some psychological support. Evidence does indeed suggest that children who have attacks tend to have higher anxiety traits.
With treatment, Professor Katelaris was clear that more clinical trials are needed in young children. They are coming, she suggested, but more needs to be done. Her priority was to get prophylactic treatment to children, as it makes a massive difference to their lives.
Women with HAE
Professor Katelaris made clear that women have more frequent and often more severe HAE attacks, with a higher need for hospitalization.
In a survey of 150 women with HAE, two-thirds stated that the condition worsened during puberty, and 80% reported more attacks if they took contraceptive pills containing estrogen. HAE attacks are often triggered by the menstrual cycle, she said.
‘Why are women so affected?’ she asked. Estrogen, a female hormone, impacts the cycle, which leads to increases in bradykinin, which, she remarked, we remember from earlier being the cause of leaky blood vessels and subsequent swelling. Monthly periods and problems such as undiagnosed endometriosis can trigger abdominal attacks.
There are oral contraceptives that do not contain estrogen, and intrauterine devices (IUDs) and barrier methods don’t seem to be an issue. In women who want to become pregnant, Professor Katelaris cautioned the need for planning. The medications the individual might be on could be a problem if they become pregnant. There is also the opportunity for genetic counseling.
Fertility is not affected by HAE, according to Professor Katelaris. Women using IVF may find that the hormonal injections can be a real trigger for attacks. Short-term prophylaxis, where available, would be good for these women, said Professor Katelaris.
Pregnant women with HAE are also diverse. In some pregnant women HAE worsens, but in others it gets better.
Professor Katelaris said that, unsurprisingly, abdominal attacks are the most frequent for pregnant women. C1-inhibitor concentrate is the only treatment recommended for women who are pregnant or breastfeeding, Professor Katelaris confirmed. There is no recommendation for routine prophylaxis before an uncomplicated birth, she said, but there should be acute treatment available regardless of the type of birth.
Breastfeeding can lead to increased attacks, and tranexamic acid and androgens are to be avoided in breastfeeding, Professor Katelaris told the audience.
Moving on to another stage in a woman’s life, Professor Katelaris discussed the impact of menopause on HAE. She reported that in 1 study of post-menopausal women with HAE, 55% of women reported no change, 13% reported improvement, and around a third reported a worsening of symptoms. In treating menopausal symptoms, Professor Katelaris expressed caution about estrogen replacement, suggesting it should be avoided and that progesterone can be used. Non-hormonal treatments are not an issue, she said.
Finally, Professor Katelaris spoke about HAE with normal C1-inhibitor, as women are far more affected by the condition than men. The face, tongue, and upper airway are usually more affected, with abdominal attacks less common. Estrogen has an even greater effect, according to the Professor. There is a lack of trials on treatment. Icatibant works but is slower, according to a case series, and there are cases where tranexamic acid has been reported to be beneficial, she concluded.
Expert panel Q&A
As the expert panel of Professor Connie Katelaris, Professor Philip Li, Dr Ankur Jindal, Dr Michihiro Hide, and Dr Jovila Abong took to the stage, Debs Corcoran, Chief Scientific Officer of HAEi, made an announcement.
She told the assembled audience of healthcare professionals, patients, and caregivers that the scientific committee had awarded Dr Jane Wong with the HAEi’s Young Researcher Award for her work submitted to the Scientific Track of the 2025 HAEi Regional Conference APAC. Debs asked the audience to give Dr Wong a warm round of applause and congratulated her on winning the award.
The panel then turned to the final, but often highly anticipated, session of any HAEi conference, when the experts respond to the questions patients and caregivers want answered.
The panel, facilitated by Dr Jovila Abong, started with the question: Hormones are usually reported as a trigger for attacks; have you noticed any trends of co-morbid hormonal disorders?
The panel noted that any imbalance in hormone levels could theoretically be a problem. Still, none of the panel felt that there was anything they had noticed in practice or seen reported in the literature.
The next question was: How can I manage my HAE while still enjoying sports?
One of the panel members disagreed with Professor Katelaris’ earlier point about contact sports. They felt that if a beloved sport is causing attacks, you can do a different sport, but you can also have prophylaxis to prevent attacks, where it’s available. If you only have non-specific treatments, this will be different, and some of the drugs, such as androgens, would lead to failed drug tests for competitors.
The panel’s main point is that we should be working towards the normalization of life. Although this might be difficult in the Asia-Pacific region right now, people should be able to play whatever sport they want with appropriate preventative medicine.
A patient question was: I need to have braces. I’m worried about an HAE attack. What can I do?
The panel’s advice was to be well prepared. In some patients, having short-term prophylaxis is good, but it depends on the extent of any dental procedures. Dental work can trigger HAE attacks. Having teeth removed is a high-risk procedure, so prophylaxis or at least on-demand treatment should be available. For braces, perhaps invisible aligners (many brands available) may be safer and should be considered case-by-case. The same advice applies, the panel said, to Botox injections, tattoos, and dermal fillers.
The next question was: Is the location of a rash an indicator of where the attack will be?
No, the panel said. The location is no predictor of an attack. You cannot predict where an attack will happen.
After that quickfire response, the next question was: When should I treat an HAE attack?
The immediate reaction from one panel member was: “As early as possible.” Guidelines do not recommend treating during prodromal symptoms; you should treat as soon as you see a lump or swelling coming, the panel said. The panel understood that the amount of medicine may be limited for some. They said that you treat every attack as soon as you recognize it in the ideal world. At the very least, always treat anything on the face or inside the mouth.
Can you cure HAE forever? was posed to the panel.
In most cases, they said, HAE is caused by a faulty gene. We don’t have a treatment to replace the gene, though that is a possibility in the future and is being looked at in trials now. That would be a one-time treatment that’s permanent. It wouldn’t prevent the faulty gene from being passed on to children.
Another treatment question was: In Europe, there seems to be an increasing reluctance to prescribe tranexamic acid due to worries about blood clots. What is your view?
The panel agreed that blood clots are a potential side effect but not a common one. The reluctance is because it’s a non-specific treatment and has limited effectiveness in many people. In Europe, they have the luxury of specific, highly effective therapies. That said, the panel agreed that tranexamic acid is the drug of choice for acquired C1-inhibitor deficiency and in people with HAE with normal C1-inhibitor.
The doctors were now asked about doctors, with the question: How do I know I am seeing the right doctor to help manage my HAE?
For the panel, the answer was a doctor who is knowledgeable but also listens to the patient. HAEi and many country-specific patient organizations have lists of good HAE doctors you can ask about. One of the panelists joked that if the patient has to ask this question, they’ve not found the right doctor.
A question was raised about access: How can I help get medication in my country?
Here, the panel said that strength in numbers was vital to access. Partnerships between patients, doctors, and the pharmaceutical industry would be beneficial. It’s no guarantee, but it is important to start trying.
The panel digressed slightly to say how important it can be for patients to motivate their doctors through their proactivity and interest. The more a patient is positive and tries to learn and help themselves, the more this rubs off on the doctor.
The panel also stressed the value of strong patient advocacy groups. This will attract research and medication, which may not immediately lead to access for all, but will raise the standard of care in a country.
A patient asked the panel: How can I explain HAE to someone new in my life?
The panel felt doctors could help, for example, by offering to speak to prospective partners. Ultimately, great information resources are available through HAEi and the Member Organizations, which can teach people a lot. Cultural concerns, like those Yong Hao raised earlier, can be problematic. The panel felt that we shouldn’t be a society where HAE is stigmatized. The panel concluded that when better treatments are available, it becomes easier to overcome any stigma.
A caregiver wanted to know how to best support a patient with HAE?
Again, the panel felt that knowledge was key. By learning about HAE, you are better able to be compassionate and supportive. It is also important to find out what the patient needs and wants. For parents, that means not smothering children out of anxiety about the condition. “We want our patients to have normal lives,” said one panelist, “we need to encourage people to make their own way in life.”
The next question asked would managing stress reduce HAE attacks, would meditation help?
The panel said that whatever works for you in relieving stress will be beneficial. Stress is the most important identifiable trigger. But it’s important to not stress about being stressed, as it’s part of everyday life. The majority of HAE attacks happen without any identifiable trigger, so if you can avoid it, do, but don’t change your life over it.
One patient asked specifically if nutrition during pregnancy could lead to HAE?
The panel said that although HAE is due to a faulty gene, there’s absolutely nothing the mother has done that would cause that. It’s not in the control of any human and is purely a random event. You should not blame the mother.
In some cultures, the panel felt there is a persistent belief that eating something or not eating something else can cause HAE. It doesn’t. HAE is entirely down to genetics.
Our final question of the session: How might perimenopause or menopause affect my HAE?
The panel referred back to Professor Katelaris’ presentation. Some women report improvement, some no change, and some worsening. There may be disturbances during this time, but after menopause, things settle down, and you adapt.
The expert panel of Professor Connie Katelaris, Professor Philip Li, Dr Ankur Jindal, Dr Michihiro Hide, and Dr Jovila Abong answered questions asked by the audience.
Thank you, and embrace the opportunities!
And with that, the conference program was complete. Tony and Henrik from HAEi returned to the stage. They thanked the expert panel for their responses. They also offered congratulations and thanks to everyone who had contributed towards the success of the meeting and particularly highlighted the support of the pharmaceutical sponsors:
Diamond supporter: BioCryst
Gold supporters: Pharming and Takeda
Silver supporters: Astria, CSL Behring, KalVista, and PharvarisBringing the meeting to a close, Tony and Henrik returned to the theme of the meeting: Embracing Opportunities. Both advocacy leaders asked those present to embrace the opportunity to work more closely together in their country and to use the tools and technology HAEi offers to achieve greater availability and access to modern HAE medicines. With those final words of encouragement, the 2025 HAEi Regional Conference APAC came to a close.
Scientific Track
Scientific track poster session
A series of submitted research posters were presented during the 2025 HAEi Regional Conference APAC. In addition to the abstracts presented during the scientific sessions, the following posters are summarised here:
Challenges of diagnosis and treatment of recurrent angioedema from Nepal- The unseen struggle in resource-constrained Settings
Dr Dharmagat Bhattarai presented work from himself and his colleagues, looking at the challenges and struggles of diagnosing recurrent angioedema. They suggest that lack of awareness and resources has negatively impacted the management of recurrent angioedema, and they report misdiagnosis, mistreatment, and poor outcomes in such resource-constrained settings.
Hereditary angioedema and cascade screening of patients from Pakistan
Dr Muhammad Hussain presented data on a cascade screening program conducted with colleagues in Pakistan. In conclusion, they felt that cascade screening facilitated the early diagnosis of asymptomatic or mildly symptomatic family members of HAE patients. This will lead to improved management, such as inclusion in compulsory vaccination programs and offering genetic counseling.
When swelling tells a story: Two unique cases of C1 esterase deficiency
Professor Ashu Sharma presented data on 2 cases: HAE and acquired angioedema. The authors concluded that when there is unpredictable swelling, timely identification and treatment can be challenging. A thorough and detailed history can reveal a hidden cause, which the authors feel can guide clinicians in their diagnosis and management.
Preliminary results from epidemiological survey of hereditary angioedema in India – just a tip of the iceberg
Dr Ankur Jindal presented data from multi-center research into the epidemiology of HAE in India. The authors suggest there remains a large number of undiagnosed patients in India, and efforts are ongoing to diagnose as many HAE patients as possible.
Acquired angioedema – Look, think, proceed!
This case report was delivered by Dr Samipa Samir Mukherjee. The focus was on a 51-year-old female with asymptomatic swelling of different areas of the body. An eventual diagnosis of acquired angioedema (AAE) was achieved. The author concluded that AAE must be considered in patients presenting with subcutaneous and/or submucosal swellings without itching or urticaria in adulthood. It was suggested that tranexamic acid is an effective form of long-term prophylaxis in patients with AAE.
Hereditary angioedema: A clinico-demographic profile of patients and availability of medical facilities in Bangladesh
Professor Bhuiyan’s research categorized patients in Bangladesh and the medical facilities available to them. The research demonstrated that there are thousands of cases left undiagnosed and missing proper treatment. The conclusions call for action to be taken to improve the capacity for diagnosis and management in Bangladesh.
Deucrictibant vs. standard of care in hereditary angioedema: A propensity score-matched analysis
Professor Connie Katelaris delivered this presentation. She described that this analysis was designed to compare outcomes of patients treated with deucrictibant and those being treated with standard of care. In conclusion, the analysis demonstrated that people with HAE treated with deucrictibant had more favorable outcomes, according to 2 methods of patient-reported outcomes, than those treated with standard-of-care treatment.
Impact of overthinking on mental health among angioedema patients: A qualitative study from Pakistan
Dr Sidra Akhtar presented their data on the psychological impact of worries and negative patterns of thought in patients with angioedema. The conclusion was that angioedema patients experience considerable mental health challenges, yet the effects of overthinking remain underexplored. The authors suggested that addressing psychological factors can enhance quality of life.
Welcome and HAE in Asia-Pacific: Country perspectives
The scientific track opened with HAEi’s Tony Castaldo spending a few minutes to set the scene. He thanked the organizing team at HAEi before introducing and thanking the Scientific Committee Co-Chairs: Professor Connie Katelaris, Professor Philip Li, and Dr Jovila Abong. He also thanked the Committee members: Dr Hilary Longhurst, Dr Hiroshi Chantaphakul, Dr Michihiro Hide, Dr Ankur Jindal, Professor Yuxiang Zhi, and Dr Felicia Racquel Salvador-Tayag. Unfortunately, Dr Chantaphakul and Professor Zhi were unable to be in Manila.
“It’s telling that none of the countries in the Asia-Pacific region have access to all the modern HAE medicines.”
Tony explained that this lack of therapeutic access is core to the mission of HAEi. “We are a fierce advocacy organization,” he told those present. He went on to outline some of the key activities of HAEi and showcased HAEi’s contribution to the science of HAE via our research programs to assist in patient identification, pharmaco-economic evaluation of therapies and quality of life, and burden of illness. Tony also drew attention to the recent HAEi and United States HAE Association Symposium and the resulting publication on HAE with normal C1 inhibitor (see our scientific publications section for more information).
Tony reminded the audience that, as clinicians, they held the key to diagnosis and treatment. He asked them to work with HAEi and their local patient advocacy organization to get access to and reimbursement for modern medicine. “It can be done,” he concluded.
On behalf of the Scientific Committee, Co-Chair Professor Connie Katelaris welcomed more than 100 participants to the scientific track and, during the opening slides, presented the Young Researcher/Investigator award to Dr Jane Wong from Hong Kong. You can read about her award-winning research in the oral abstract presentations.
The scientific program started with a session dedicated to country perspectives on HAE. The 3 countries represented were the Philippines, Malaysia, and Nepal. Dr Ankur Jindal chaired the session, and the first presenter was Dr Jovila Abong, who spoke on the state of diagnosis and management of HAE in the Philippines.
HAE in the Philippines
Dr Abong opened by stressing that a lack of data in the Philippines hinders proper diagnosis and management of HAE and that reported cases are clustered around diagnostic testing sites. In 2024, a survey was conducted amongst members of the Philippine Society of Allergy, Asthma and Immunology (PSAAI). The 88 respondents reported 14 confirmed cases and 28 suspected cases. The initially prescribed medications were mainly allergy medication, and most suspected cases were treated as allergies. Dr Abong described the geography of the Philippines, with 7,641 islands divided into 3 regions: Luzon, Visayas, and Mindanao. Dr Abong indicated limited access to HAE testing facilities in Visayas and Mindanao, but more in Luzon where Manila is located.
Moving on to treatment, Dr Abong explained, that in the Philippines, on-demand treatment is fresh frozen plasma. Additionally, tranexamic acid and danazol are available. Patients with relatives and friends outside the Philippines can acquire other drugs such as C1-inhibitor infusions.
Looking to the future, Dr Abong indicated that PSAAI is working to include HAE in the Philippine’s Rare Disease Act. It would unlock access to government funding for diagnostic tests and treatment if accepted. Dr Abong mentioned ongoing dialogue with pharmaceutical partners, with an application for compassionate access being developed. Dr Abong felt hope in collaboration with patients and caregivers on activities to improve awareness, diagnostics, and access to treatment.
HAE in Malaysia
Malaysia, according to Associate Professor Adli Ali, was the youngest member of the HAE community in the Asia-Pacific region. The first reported HAE cases were in 1996, but in the subsequent years, treatment remained limited to ‘medieval treatments’ such as fresh frozen plasma. Today, Associate Professor Ali indicated that on-demand medication with icatibant is available through emergency protocols.
Currently, Malaysia has 22 diagnosed patients, most with HAE Type 1. Associate Professor Ali reported that there is a pool of interesting novel mutations amongst Malaysian HAE patients. There are immunologists with knowledge of HAE, but most of these are pediatric specialists. C4 complement testing is available at all centers in Malaysia. Quantitative and functional tests are available but require patients to pay.
Associate Professor Ali highlighted that there is now an HAE working group, with clinicians, mainly immunologists and dermatologists, and they are developing a national guideline to give a framework for diagnosis and management. Hopefully, this will create awareness and conversations among the HAE community in Malaysia.
Overall, Associate Professor Ali told the audience there is a need to improve awareness and understanding, so an education module is in development. Doctors in Malaysia are also actively developing a national guideline. Finally, Associate Professor Ali confirmed that centers in Malaysia look forward to being a greater part of the ACARE community.
HAE in Nepal
The last country perspective was given by Dr Dharmagat Bhattarai, who is from the beautiful and mountainous country of Nepal. Based on the prevalence studies, there should be more than 50 HAE patients, but this number has not been diagnosed, and the lack of affordable testing facilities hampers diagnosis.
Dr Bhattarai recounted how he established his center without support from the Government or other organizations, and it is dedicated to awareness and advocacy for immunological diseases such as HAE.
Testing facilities are an issue, as there are no genetic testing facilities in Nepal. All genetic testing is sent to a commercial lab in India. In terms of management, Dr Bhattarai referenced that the situation in Nepal is similar to Malaysia and the Philippines, in that most historically diagnosed patients were treated as allergy cases with antihistamines and steroids.
The current landscape of treatment is primarily based on the use of tranexamic acid and steroids such as danazol or stanazol. Acute attack patients are treated with fresh frozen plasma. Dr Bhattarai noted that 2 of his more fortunate patients can purchase modern medicine from India. Some medications have also been donated from institutes in Europe. Dr Bhattarai characterized the situation as ‘people are pleading for treatment.’ He has worked hard to raise awareness and advocate for the disease. Positive recent steps have included the creation of HAE Nepal as a patient organization. There are also plans to register C1-inhibitor concentrate and ensure genetic testing facilities, and he thanked all those who have helped Nepal to date.
Panel discussion
Following the third perspective, there was a panel discussion and audience Q&A. The first question was on the use of tranexamic acid. The panel believed that tranexamic acid should be used only in the absence of alternatives. After this, there was a discussion about problems with registering C1-inhibitor infusions in Nepal. Dr Bhattarai suggested the situation was due to a cumbersome registration process and a lack of engagement with the scientific literature. There is also a lack of awareness and understanding of the need for treatment, as HAE is a life-threatening disease. He also suggested that there is a focus on medicines for more common diseases. Ultimately, he suggested that the onus for completing a registration fell to him.
Having heard about a national database in Malaysia, a doctor from India asked about avoiding data duplication in the actual number of HAE patients. Associate Professor Ali responded that there is inevitable overlap but that this can be helped by making someone the gatekeeper of the data. Duplication is not such a problem in Malaysia right now as there are few patients, and Associate Professor Ali knows the names and ages of many of them. Dr Hide gave a Japanese perspective, as the country has 2 registries. For the patients themselves, there is no duplication, but there may be multiple entries by different physicians, which requires addressing.
The final question was about the most important things to do to make the medication accessible in a country. The panel highlighted the value of an orphan drug registration pathway where it exists. The next step was to focus on need. The panel commented that on-demand therapy may be most desired when speaking to patients, and, therefore, the focus should be there.
The advocacy of patient organizations, locally and through HAEi, was also seen as a positive step. The importance of increasing diagnosis was stressed, as having a defined patient population demonstrates the need for treatment.
Oral abstracts session 1
The next session was the first of 2 sessions in which submitted abstracts were presented. In this session, chaired by Dr Michihiro Hide, 3 abstracts were presented:
- Unearthing hereditary angioedema in India: Epidemiology from Chandigarh and Reasi
- Evaluating the effectiveness of cascade family screening among Sri Lankan hereditary angioedema (HAE) patients
- Screening program providing outreach for testing hereditary angioedema (SPPOT-HAE): Validation and utilizing dried blood spots for family screening
Unearthing hereditary angioedema in India: Epidemiology from Chandigarh and Reasi
The first presentation, on epidemiology from the Indian regions of Chandigarh and Reasi, was given by Dr Suprit Basu. Dr Basu appeared via an online link due to an emergency situation at his hospital.
He outlined that the research aimed to highlight the challenge in diagnosing and managing HAE in resource-constrained settings by addressing the limited epidemiological data on the disease in developing countries.
Dr Basu and team began by reviewing records of all patients diagnosed with HAE between 1994 and 2023. They then conducted 2 field surveys to trace further potential patients and calculate the prevalence of HAE in Chandigarh and Reasi. 12 patients from 6 families were found in Chandigarh. In Reasi, 41 patients from 5 families were uncovered. The prevalence in Chandigarh was 1 per 100,000 population, but the rate in Reasi was 10 times this.
Dr Basu reported that all patients were HAE Type 1 in the Chandigarh cohort. All of the Reasi patients were Type 2, with laryngeal attacks and tongue swelling being more common in this group.
In conclusion, Dr Basu suggests that there is an urgent unmet need to diagnose all patients. In a brief Q&A, he clarified further that the different prevalence rates may be due to large inter-ethnic differences. The stated prevalence in these areas is the minimum, as awareness of the disease is so low in Chandigarh and Reasi. More education would increase diagnoses.
Evaluating the effectiveness of cascade family screening among Sri Lankan hereditary angioedema (HAE) patients
Speaking next was Uvini Amarasekara. She echoed Dr Basu’s work by stating that there is no consensus on HAE prevalence in Sri Lanka, with many cases remaining undiagnosed. Part of this, she said, was due to a lack of immunologists and little funding from authorities for treatment or research.
The research presented focused on 20 confirmed patients with HAE. Through family screening, 98 family members were identified, of which 28 were symptomatic but not diagnosed with HAE. Through diagnostic testing, 12 new cases of HAE were diagnosed in these 28 patients. Diagnosis rates, quality of life, and angioedema activity scores were calculated for all patients identified. The quality of life and activity scores suggested some impact on the lives of people with HAE.
In follow-up questions, Ms Amarasekara suggested that the barrier to diagnosis is a lack of awareness of the dangers of HAE. When patients are educated about the condition, they are willing to bring their whole family for screening.
Screening program providing outreach for testing hereditary angioedema (SPPOT-HAE): Validation and utilizing dried blood spots for family screening
The last presentation was given by Dr Jane Wong, who received the HAEi Young Researcher award for this scientific work.
She indicated that funding for the research was from Takeda. By way of background, she told the audience that many laboratory assays are available to diagnose HAE, but these are not readily available, especially in rural areas. In particular, she highlighted the laborious nature of C1 function testing, as samples must be tested within 2 hours and kept on dry ice. All of this is a major barrier for a lot of centers.
The concept of dry blood spot (DBS) is one potential solution to this issue. It is an existing technology, and dry blood spot has been adapted to test for C4 and C1 function and C1 inhibitor level. DBS is very stable, Dr Wong told the audience; DBS does not need to be transported on ice and it can be put in an envelope at room temperature.
“For SSPOT-HAE, you only need five drops of blood. And you can put it on your desk for a week before the FedEx people come to take it.”
This research was designed to validate if DBS compares with conventional laboratory testing for HAE. Patients with known HAE were recruited to come to the clinic and be tested using DBS. Additionally, previously untested relatives of these patients were also tested with DBS (followed by conventional lab methods). The DBS testing corroborated well for the previously diagnosed HAE patients, Dr Wong said. The DBS also seemed to encourage previously reluctant family members to attend for treatment. 9 patients in one family were screened; 2 were ultimately confirmed to have HAE.
In conclusion, Dr Wong suggested that the testing had identified no false negatives, which was a concern as it would mean patients with HAE would be falsely told they did not have the condition. Her key message, she said, was that DBS can be a good tool for family screening and be used in outreach programs to overcome some of the barriers to wider testing, such as logistics concerns inherent with existing laboratory tests.
The follow-up questions focused on the issue of false positives. Dr Wong acknowledged this is a possibility, but their concern was more with false negatives, as this would put people with HAE at risk of being untreated during attacks. Dr Wong clarified a question about blood sampling affecting the test; if there is enough blood to fill a spot on a paper, it can be tested.
The Patient Journey and HAE Management
The third session of the scientific track was chaired by Dr Hilary Longhurst and devoted to the patient journey and HAE management.
The Patient Perspective
Taking a break from his role as an HAEi Regional Patient Advocate, Yong Hao shared his experience of life with HAE.
He started by telling the physicians and scientists present that his first attack took place when he was 21, just as he was about to begin his national service in the army. He remembers being confused as he had hurt his ankles before playing basketball. This time, the sole of his right foot was swelling more and more. After a few days, he went to the emergency department at the urging of his family. He was admitted to the hospital, where they thought he might have gout or arthritis. He was discharged when the swelling started to subside but without a diagnosis.
Yong Hao used the term ‘diagnostic odyssey’ to describe his experience, which he suggested was common with others in the region. He saw numerous doctors of different specialties in different hospitals before finally being diagnosed. He said that for some people, this can lead to losing trust in medical professionals as no one seems to know the problem.
Additionally, Yong Hao felt there may be a problem for patients and family members who have not yet been diagnosed and are unwilling to follow up with a doctor. Some of this, Yong Hao suggested, can be due to cultural concerns about becoming a burden on their family but also the idea that ‘Western’ medicine may not be effective and may have side effects.
Yong Hao described a breakthrough in diagnosis when he started to keep a record of his attacks and to take photos of the swelling, especially since he had never seen a specialist while having an attack. After seeing the images, the specialist was able to rule out gout or arthritis. Yong Hao also searched online once he was able to exclude those 2 conditions and saw information about HAE, which he was able to discuss with his specialist. It led to testing, and 2 weeks later, a confirmation of HAE Type 1.
Yong Hao told the audience he is still treated by this specialist, who is still unfamiliar with the condition but follows guidelines on the management of the condition. He receives danazol for preventative purposes and C1-inhibitor for on-demand treatment. He stressed to the audience that, as a patient, it is easy to trust your doctor when your condition is well-managed.
According to Yong Hao, one way to build trust with patients is to have a bigger conversation than just diagnosis and treatment. Asking about the impact of HAE on finances, work, and social life is important for patients to feel that their doctor cares about their experience. By switching from a curative conversation to one that focuses on understanding the individual, patients feel better able to trust. However, Yong Hao felt that healthcare systems are rarely set up to achieve this.
In conclusion, Yong Hao urged physicians to tailor consultations and conversations to fit the needs of HAE patients, allowing for issues beyond treatment to be discussed. By better understanding the experience of patients and by working with patient advocacy groups, clinicians can build trust and better manage the condition.
HAE: Beyond the physical symptoms
Debs Corcoran from HAEi was joined onstage by Krizzia Lavone Ramos Alferez from HAE Philippines. Introducing the session, Debs reminded the attending physicians that while the physical symptoms of HAE may be obvious, as they can be seen and felt, HAE has a broader impact on those living with the condition and their families, which is often not seen and not talked about. Debs then turned to Krizzia to ask her about her journey with HAE.
Krizzia told the audience that she and her family were the first HAE patients to be diagnosed in the Philippines. She was diagnosed by Dr Abong, who spoke in an earlier session. Krizzia’s HAE started when she was 14 years old. She had swellings in her hands and feet, and other members of her family had similar symptoms, but these were dismissed as just sensitivities. After her mother was hospitalized with a throat swell, they were all tested and diagnosed with HAE.
Before her diagnosis, Krizzia remembers having to miss lots of school, college, and work. She has now worked closely with HAEi to build HAE Philippines as a patient organization and collaborates with doctors on helping patients take action.
Debs asked Krizzia to tell the audience more about how HAE makes her and other patients feel, especially when it causes them to miss school and work.
Krizzia said it’s not the same for everyone, but attacks are painful, so people can’t do their everyday work, which means they miss out and find it hard to catch up. She also said that the worry HAE causes can make people miss out on many things. She’s heard people say they don’t want to travel in case they have an attack, which means they miss out on social activities, holidays, and even seeing family. Being away, Krizzia said, is also a source of worry for caregivers. Caregivers often talk about feeling guilty if they are away and a family member has an attack.
Debs then asked Krizzia to tell the doctors present how they can help patients with the broader impact of HAE. Krizzia responded that she’d like them to consider more than just the physical symptoms. Asking patients, ‘How are you feeling?’ could be an important question. As a nurse, she knows that asking such questions can be hard as there might not be a simple answer, but for patients and being a patient, too, just being asked can be really helpful. She said simple self-care can help people feel less anxious or worried.
Having a knowledgeable doctor who can support you is also a great relief for patients. Krizzia suggested that even if treatment options are limited, knowing your doctor understands HAE helps. An important part of this is working with Member Organizations to ensure good educational materials for doctors, patients, and caregivers.
In conclusion, Debs thanked Krizzia for her bravery in discussing these issues. She also highlighted the resources currently available on the HAEi website. Additionally, she trailed a forthcoming HAEi leaflet on individual self-care wellbeing, which will be available as part of preparations for hae day :-) 2025.
The Physician Perspective
Rounding out the session was Professor Philip Li, who gave his own experience of the importance of understanding and appreciating the lives of his patients.
He opened by saying that for him, really listening to patients is one of the most rewarding parts of his daily practice and that by doing this, it’s possible to know that a difference has been made to an individual.
Professor Li told the audience a story about an imaginary world in which every patient was diagnosed quickly and without problems relating to cost and availability of testing. The individual would be referred to him fast and would understand and accept their diagnosis. Depending on their disease and personal preferences, a range of treatments would be available for that patient. After the third visit, the patient’s HAE would be conquered!
“This has never happened – yet – in my career,” was Professor Li’s assessment. The facts are that he is still looking for patients; many are unwilling to be tested, medications are limited or not available at all, regional data is lacking, and stigma remains for people with HAE.
All of the problems, according to Professor Li, can be overcome if physicians work with and empower their patients. To raise awareness, Professor Li told the audience, “I’m completely powerless. I 100% depend on patients. I’m boring, but a patient sharing their story is different.”
The situation is similar to issues of medical education. Patients’ contributions can develop the materials needed, and by sharing them widely with their doctors or those they see in emergency rooms, the knowledge of HAE will increase.
One big collaboration area for Professor Li is increasing the number of diagnosed patients. Cascade family screening is beneficial. Professor Li suggested speaking to existing patients to uncover potential cases, especially as Asian families can be huge. For him, this was crucial as increasing the population of patients means expanding the interest of pharmaceutical partners. “You need a critical mass”, he told the experts present.
He acknowledged that it is easy to be discouraged and demotivated.
“We have all been there. Every one of us has felt it’s impossible.” Professor Philip Li
Professor Li stressed that the important thing is to collaborate: Work with your patients and colleagues.
In the subsequent Q&A session, Professor Li discussed the ultimate diagnosis in children of asymptomatic parents. He suggested it is difficult to give exact answers as it depends. It can be that a patient is not asymptomatic but had forgotten a solitary attack some time ago. He cautioned that doctors should challenge patients to ensure they can tell you everything you need to know.
Finally, Professor Li also urged his colleagues across APAC to work together on a plan to improve the available local and regional data, as this will help develop appropriate regional guidelines. The first step for him would be to understand the number of diagnosed patients receiving care.
Professor Li stressed that the important thing is to collaborate: Work with your patients and colleagues.
Oral abstract session 2
The fourth session of the scientific track at the HAEi Regional Conference APAC returned to the oral abstracts and chair Dr Michihiro Hide. This time, 2 research studies were presented:
- Identification of candidate biomarkers for diagnosing type-2 hereditary angioedema and assessing disease severity via plasma N-glycomics
- Long-term safety and efficacy of oral deucrictibant for prophylactic and on-demand treatment of hereditary angioedema attacks: Results of the ongoing CHAPTER-1 and RAPIDe-2 extension trials.
Identification of candidate biomarkers for diagnosing type-2 hereditary angioedema and assessing disease severity via plasma N-glycomics
Dr Xiangyi Cui from China gave her presentation via video. She told the audience that the study’s background is that the current HAE test can be limited due to instability and challenges with sample handling, storage, and transport. She also noted that there are currently no biomarkers to assess disease severity in HAE Type 2, limiting prophylactic treatment optimization.
Early work, highlighted by Dr Cui, indicated that N-glycans could diagnose and assess the severity of HAE Type 1. This study aimed to explore the potential of plasma and glycan biomarkers in HAE Type 2.
Samples were collected from patients with HAE Type 2, mast-cell angioedema, and healthy controls. These were analyzed, leading to 109 N-glycans being identified in total and 63 passing quality control procedures. Several N-glycans were identified that were significantly different in HAE Type 2 patients and healthy controls. Of these, 2 N-glycans were significantly associated with disease severity.
Dr Cui concluded that these novel biomarkers show significant potential for diagnosing HAE Type 2, predicting edema size, and assessing disease severity. She told the audience that the team working on this research is excited about the potential.
Long-term safety and efficacy of oral deucrictibant for prophylactic and on-demand treatment of hereditary angioedema attacks: Results of the ongoing CHAPTER-1 and RAPIDe-2 extension trials
Despite being from Bulgaria, Associate Professor Anna Valerieva was a welcome addition to the Asia-Pacific region. She presented data on this potential new treatment on behalf of her co-authors.
Associate Professor Valerieva stressed that deucrictibant remains an investigational agent that is not currently licensed for use. It is being trialed for both on-demand and prophylaxis of HAE attacks. Associate Professor Valerieva characterized the current prophylactic therapies are having some drawbacks, and there is a need for a treatment that is easily administered with efficacy as good as injectable therapies. Deucrictibant is an oral agent. An immediate-release capsule is given as on-demand therapy, while an extended-release tablet is being tested as a prophylactic agent.
In both on-demand and prophylaxis studies, Professor Valerieva characterized the safety data as very encouraging. There were very few adverse events considered to be related to treatment and no treatment discontinuation.
In terms of efficacy, Associate Professor Valerieva suggested they are promising. The studies showed reductions in attacks. For prophylaxis, this reduction happens within 1 to 2 weeks of drug initiation. The reduction translated to a 14-month attack-free period for patients in the study. In the on-demand trial, there is a rapid reduction in the severity of attack symptoms, typically 2.6 hours (median time). 98.5% of patients experienced the start of symptom relief within 12 hours.
In conclusion, Associate Professor Valerieva said the studies provide evidence for the long-term safety and efficacy of deucrictibant. It is also well tolerated, Associate Professor Valerieva told the audience.
Some asked Professor Valerieva of the audience about the oral nature of the medication and its use in abdominal attacks where patients may be vomiting. She suggested that the absorption of the medicine is very rapid, about 5 to 10 minutes. Her view was that if the patient can keep the pill down for that long, it would work. There were also quite a lot of abdominal attacks in the study. The size of the pill was also asked. Professor Valerieva suggested it is no larger than a regular pill, such as an antibiotic. The availability of the medicine, or a liquid formulation, for children was also discussed, and trials of the drug in children are planned.
Clinical case presentation, discussion, and close of the Scientific Track
Dr Felicia Racquel Salvador-Tayag chaired the final session in the Scientific Track. It featured a short case presentation by Professor Connie Katelaris and a discussion with a panel from the Scientific Committee.
She told the audience about an otherwise healthy-looking young boy who regularly presented at the emergency room with recurrent, severe abdominal pain. Despite extensive tests, IV fluids, and pain relief, many theories were suggested, but no concrete cause or diagnosis was made.
Eventually, after being admitted to a hospital ward for observation, the boy had an attack that was observed and he was diagnosed with HAE Type 1. Treatment at the time was limited to tranexamic acid and C1-inhibitor for severe attacks.
At a much later date, when the boy had become an adult, it became apparent that his father, who had no contact with the family, knew that he had a rare swelling disorder but had never disclosed that to his ex-wife, who cared for the boy.
Professor Katelaris made clear that the boy and a sibling suffered because of that non-disclosure. She felt that the lessons from this are many around differential diagnosis and reiterated something Professor Li had said previously, that it is imperative to track down the family. “Sometimes, it’s impossible due to family dynamics, but we must try all we can,” she said.
An audience Q&A and discussion followed this. At the conclusion, the Co-Chairs drew the Scientific Track to a close. They thanked all speakers, participants, and audience members. After lunch, the physicians and scientists would join the main patient track for the final presentations.
On Saturday afternoon, the 3 tracks came together for the eagerly anticipated talks and Q&A with HAE experts from the 2025 HAEi Regional Conference APAC Scientific Committee.
Youngsters Track
APAC youngsters – advocacy, inspiration, and fun!
In common with HAEi Regional Conferences in EMEA and the Americas, young people from across the Asia-Pacific region gathered in Manila for a specifically tailored program of advocacy, activities, and fun, led by Nevena Tsutumanova, Manager HAEi Youngsters’ Community and Special Projects.
The Youngsters Tracks are closed, safe spaces for young people to gain knowledge, ask questions, and, most importantly, build friendships with HAE patients and caregivers – just like them. In Manila, 25 patients and caregivers from 7 countries participated in networking and fun.
Nevena told Global Perspectives that all Youngsters Tracks start slowly, with young people unsure what to expect. “I see the hesitation on their faces,” she said. But that nervousness soon disappears, and Nevena continues: “A lot changes in a short time. The same faces light up with smiles. People exchange contacts and chat over coffee.”
“The Youngsters Track opens peoples’ minds to different possibilities.”
Nevena TsutsumanovaPlanning the program
The Youngsters Track at any HAEi Regional Conference is developed in collaboration with the HAEi Youngsters’ Community Youngsters’ Advisory Group (YAG), to ensure that all the sessions and content are relevant and useful to the young people participating. We included sessions about HAE and wellbeing and prioritized a more relaxed, facilitated discussion session to make it easier for youngsters to share their experiences – a critical part of the HAEi Youngsters’ Community.
Hearing global stories
Of course, as the name suggests, this regional conference was focused on the Asia-Pacific region. However, Nevena knows that many of the issues young patients and caregivers face are similar across the globe. To help facilitate conversations, the team collected a series of stories from across the HAEi Youngsters’ Community and shared them with the group in Manila. The stories provided a potent way to start conversations, even on tricky topics like resilience and overcoming stigma. Nevena told Global Perspectives: ‘It was wonderful to see the engagement these stories provoked and the questions that followed.”
“To not let HAE hold me back from anything is what I took away.”
Youngsters Track ParticipantThe power of stories
You might remember Faye Marshall from earlier editions of Global Perspectives. This time, our resident word wizard joined Nevena in Manila. She helped the young people tell their stories and express themselves through the art of words. A series of workshops and discussions helped young people to express their thoughts and feelings. Nevena had high praise for Faye, calling her “A rockstar and a magician. She was outstanding, and we hope to collaborate again.”
Young people’s HAE questions answered
Dr Hilary Longhurst, a member of the Scientific Committee of the 2025 HAEi Regional Conference APAC, took time out of the Scientific Track to answer the burning questions of young people. As in previous meetings, this is always a popular part of the agenda. Young people from the Asia-Pacific region asked about the future, being an adult, relationships, and starting a family, and the present and future of treatment for children with HAE.
“The young people were grateful to Dr Hilary Longhurst for her time and thoughtful answers. She created a safe space for the youngsters.”
Conclusions
The feedback received tells us the track was a real success with these APAC Youngsters. The YAG’s contributions in the planning stages ensured it met young people’s needs. With each Youngsters Track, we open minds to the possibilities of working together as a community and the importance of advocacy. The young people who attended produced many images, videos, and postcards, and we will use them throughout the HAEi Youngsters’ Community in the coming months. Nevena also heard from some of the attending youngsters that they wanted to get more involved in HAEi and advocacy, so watch this space!
“I will use the Youngsters Track information to be a great advocate for myself as a HAE patient and for others as well.”
Youngsters Track Participant
On Saturday afternoon, the 3 tracks came together for the eagerly anticipated talks and Q&A with HAE experts from the 2025 HAEi Regional Conference APAC Scientific Committee.
We’re only a few weeks away from 16 May and hae day :-) where we celebrate the HAE community and focus on awareness-raising efforts.
So far, we’ve had more than 270 images posted to our gallery and more than 3,900 activities recorded since the activity challenge opened on 1 April. We’ve made some changes for 2025; read on to learn more!
Our #active4HAE activity challenge goes purple!
The #active4HAE activity challenge is back – but with a purple twist! We continue encouraging everyone to participate in physical and wellbeing activities and to use purple to show support. You can wear purple clothing or accessories, use purple equipment, spot purple in your activity environment, or get creative with anything purple during your activity!
Why purple?
Purple is one of the colors in our hae day :-) logo and has been adopted by many HAEi Member Organizations (MO) as the color they use in their activities for hae day :-) such as releasing balloons or lighting up buildings. It is also a color associated with rare diseases, as it is one of those used by Rare Disease Day.Share your Insta story with our stickers
If you share your hae day :-) or #active4HAE pictures in an Instagram story, you can add one of our new stickers. Search for HAEi when creating your story to find and add the campaign GIFs.Introducing a Personal Activity Goal in your profile
If you have a profile, you can now set a personal target for the number of activities you want to complete during the challenge. Each time you log an activity, it counts towards your goal and helps you stay consistent. Your profile’s new totalizer feature shows your progress – and you earn a new badge once you’ve hit your target!All the features you know are still there
In your profile, you can earn badges by logging activities each week of the challenge and choose to support your Member Organization team, HAEi, or Youngsters. Each activity logged will contribute to your country’s total; anyone can add photos to our gallery. You can download #active4HAE image assets from our website to use in photos you share.>> Share your awareness activity or event on our calendar page
Head to haeday.org to find out more!
Join the challenge and help us travel 3.5 times around the world and beat last year’s total!
At the end of April, our 18 HAEi LEAP Class of 2025 students will head to Copenhagen for the 3-day in-person kick-off meeting.
Nevena Tsutstumanova, HAEi’s Manager, Youngsters’ Community and Special Projects, and Debs Corcoran, HAEi’s Director Research, will take the students through a packed program of learning, connecting, and creating memories. We look forward to welcoming Dr. Carolina Vera from the Institute of Allergology at Charité – Universitätsmedizin in Berlin for a special “Face2Face with an HAE Expert” session.
After the in-person meeting, the students will continue their education online before putting everything they’ve learned into practice by working on an advocacy project with their Member Organization.
You can follow the progress of the class of 2025 here.
Stay tuned to Global Perspectives as we bring you all the news from the kick-off meeting and beyond in future editions.
Nevena and Debs
We are delighted to have the support of the following pharmaceutical companies for the HAEi LEAP 2025 program, who will join us in Copenhagen. Thank you.
Update from Dr Jindal in India and his new role on the ACARE Steering Committee
Dr Ankur Jindal is Assistant Professor of Pediatric Clinical Immunology and Rheumatology at the Allergy Immunology Unit, Department of Pediatrics, Postgraduate Institute of Medical Education and Research in Chandigarh, India. He is a key part of the HAE community in India; his center is a certified ACARE (Angioedema Centers of Reference and Excellence), where he is the department head. He is the General Secretary of the Hereditary Angioedema Society of India (HAESI) and a strong supporter of HAE India. In addition, Dr Jindal was recently elected to the Steering Committee of ACARE.
Global Perspectives (GP) was fortunate to get time with Dr Jindal at the fringes of the 2025 HAEi Regional Conference APAC, where he was part of the Scientific Committee. In addition to asking about his work with ACARE, which is a joint collaboration between HAEi and the Global Allergy and Asthma Excellence Network (GA2LEN), GPs asked about the advances in HAE in India and what this could mean for patients with HAE in other resource-constrained settings.
GP: Congratulations on becoming part of the ACARE Steering Committee. What does this mean for you?
Dr Jindal: Thank you so much. It is indeed a big honor for me to be a part of the ACARE Steering Committee. ACARE is the largest and strongest global network of centers and physicians who not only provide care to patients with angioedema and hereditary angioedema but are also actively involved in clinical trials and research. ACARE also provides a unique platform and opportunity for physicians to initiate collaborative research with more than 100 centers in the network.
Being part of this robust network has always been a dream of mine. Our Institute has been an ACARE center since 2021, which has been enormous for us in advancing the care of patients with HAE in India.
Becoming a part of the ACARE steering committee, one of the organization’s most important decision-making units brings responsibilities to advance progress in clinical, diagnostic, and treatment-based research.
GP: What do you hope to bring to this new role?
Dr Jindal: I represent a part of the world where diagnostic and management facilities for angioedema and hereditary angioedema are not fully developed or easily accessible. Hence, I bring a perspective from a ‘resource-constrained’ setting to the network. I am looking forward to my new role as an ACARE Steering Committee member.
GP: Can you give us a brief description of the current situation for HAE in India and any changes or improvements on the horizon?
Dr Jindal: HAE in India has been full of challenges but also unique opportunities. A major challenge for us is to improve awareness of HAE amongst physicians and to improve the diagnostic rate of HAE. We have accomplished this goal through Continuing Medical Education (CME), webinars, and publications in several parts of the country. Approximately 50-100 new HAE patients are diagnosed annually in India. However, we are nowhere near diagnosing all we might expect to have HAE, an estimated 27,000 to 135,000 patients. Although the diagnostic facilities for HAE have improved significantly, it is an ongoing process.
Another major challenge is the availability and access to first-line treatment options. Plasma-derived C1 inhibitor concentrate has been available in India for over 2 years. Through the national rare disease policy of the Government of India, we have been able to provide access to plasma-derived C1 inhibitor concentrate to some of our patients, and several other patients will soon be able to access this treatment. We anticipate clinical trials from 2 pharmaceutical companies to start in India in early 2026.
We established a physician society for HAE, the HAE Society of India (HAESI, www.haesi.in), in February 2021. Since its inception, the society has taken several critical steps towards improving awareness of HAE and bringing first-line treatment options into the country.
We also have a registered patient society for HAE (HAE India), which is actively working to improve patients’ quality of life and awareness about HAE in the country.
GP: Global Perspectives understands you do a lot to support countries in your region. Can you tell us more about this work and the impact it has had?
Dr Jindal: Having understood the challenges for HAE in India and the ways to overcome many of them, we started recognizing similar challenges in several South-East Asian countries as well.
In collaboration with HAE International (HAEi) and the Bangladesh Dermatology Society (BDS), we organized an educational webinar for physicians in Bangladesh. We followed this with a dedicated 5-hour HAE session at the national BDS conference in July 2024. As a result, a dedicated Urticaria/Angioedema-focused group of physicians formed in Bangladesh. This group works with the Bangladesh patient organization to improve HAE awareness.
We invited representatives from Nepal, Sri Lanka, Malaysia, and the Philippines to the ACARE Preceptorship in Goa in late 2024. Representatives from Nepal and Kazakhstan also attended the ACARE Preceptorship held in Chandigarh in February 2023. These medical education events significantly improved HAE awareness in these countries.
We have also organized CME for dermatologists in the Philippines in collaboration with the Philippine Academy of Clinical and Cosmetic Dermatology (PACCD) in March 2025. If we can secure support, we hope to continue educational activities for HAE for physicians in the Philippines.
GP: What would you say to other healthcare professionals seeking to replicate your success in diagnosing patients?
Dr Jindal: Our beloved Dr Marcus Maurer always taught us: ‘Never Give Up.’ Despite several challenges in India, we never gave up. We must utilize the available resources as best as possible, but we should never stop trying to get something better for our patients with HAE.
In countries where first-line treatment options are not easily available or accessible, it is common for HAE patients to lose hope and accept this disease as part of their lifestyle. However, as physicians, we must reach out, keep motivating, and provide hope. And do this in collaboration with the patient organization in your country/region.
Global Perspectives: How important is a partnership with patient advocacy organizations to your work in HAE?
Dr Jindal: I still remember the first-ever patient-doctor meeting HAEi organized in New Delhi, India, in March 2019; it was a stepping stone, and we never looked back. The support from HAEi and HAE India has been phenomenal in our efforts to improve awareness and bring first-line treatment options for HAE in India. HAE patient advocacy organizations have always been an integral part of our efforts and continue to do so; they are essential to the activities we carry out.
The HAESI recently organized 2 healthcare professional education meetings on 23 and 24 March 2025 in Odisha (a coastal state in the eastern part of the country). We always include a talk on patient perspectives, and listening to their journey taught the physicians who attended more than disease facts can convey.
The presence of HAEi acts as a launch pad to drive your efforts to improve awareness and bring first-line treatment options for HAE in their countries. I always convey this message to my colleagues from neighboring countries. Now, we have real-world evidence to demonstrate HAEi’s impact. A recent publication from the Asia-Pacific region showed that HAEi patient group presence in a country significantly improves the diagnostic rates of HAE and access to first-line treatment options.
– – – – – – – –
Global Perspectives is grateful to Dr Jindal for sharing his work in HAE with us, especially at such a busy time. Dr Jindal is a powerful advocate and voice for resource-constrained countries. His election to the ACARE Steering Committee can only help ensure the needs of this vitally important patient population are heard, understood, and acted upon. You can learn more about ACARE’s work at haei.org and acare-network.com.
Have you wondered how to get your HAE under control? HAE TrackR can help you!
Developed by fellow HAE patients at HAEi, HAE TrackR is an easy-to-use electronic diary designed to record your HAE treatments, HAE attacks, and the impact HAE has on your life and the lives of your loved ones.
HAE TrackR allows you to download a comprehensive report of your treatments and attacks, which can be used as a tool for you and your physician to manage your HAE.
Features of the app:
- Records your treatments (preventative, on-demand and clinical trial medication)
- Records attacks and developments/improvements of attacks
- Has a smart reminder functionality for prophylactic treatments
- Is endorsed by ACARE for its ease of collecting and using data and comprehensive reporting
- Is safe and secure, product and company neutral with no commercial interests
News from HAEi Countries Around the Globe
From HAE Armenia
We are extremely happy to announce that the study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in patients with congenital C1-INH deficiency is now open at the Heratsi Hospital in Yerevan. This study is the opportunity for HAE patients to access a modern investigational treatment option, and hopefully, more clinical studies in Hereditary Angioedema in Armenia will be followed.
From Zoe Steiner, Operations Coordinator, HAE Australasia
HAE Australasia Update: A Busy Start to 2025
It’s been an action-packed start to the year for HAE Australasia, with significant events and initiatives taking shape across the region. We’ve been hard at work supporting and advocating for the HAE community, ensuring that our members across Australia and New Zealand are well represented and supported.One of the major highlights of the year so far was the 2025 HAEi Regional Conference APAC, which took place on the 8th and 9th of March in Manila. We were thrilled to see a strong representation from Australia and New Zealand, with many of our members featured on stage, sharing their own patient stories and taking part in insightful panel sessions. It was inspiring to witness the depth of knowledge and passion for HAE shared by all involved. The Youngsters Track was also a standout, with our young people having a fantastic time. The HAEi team did a remarkable job organizing the event and ensuring everything ran smoothly, and we extend our heartfelt thanks to them for their efforts in making it such a memorable experience.
Looking ahead, we are excited to announce our HAE Australasia 2025 Patient & Carers Meeting New Zealand, which will take place on the 3rd of May in Auckland. Invitations to register have already been sent out, and we’re in the final stages of fine-tuning the agenda. This meeting promises to be an essential event for our New Zealand HAE community, offering valuable guidance and a chance to discuss key issues affecting our members. One of the primary topics will be how we can strengthen our advocacy efforts in New Zealand, pushing forward our mission to ensure that prophylactic care becomes a standard option for all HAE patients. We expect this event to draw a strong turnout and provide a great opportunity for the New Zealand HAE family to come together and collaborate.
As always, we’re deeply committed to supporting our HAE community, and we look forward to continuing our work to improve the lives of patients across Australia and New Zealand in 2025 and beyond. Stay tuned for more updates and exciting developments!
From Raquel de Oliveira Martins, HAE Brazil/Abranghe
On March 10, the São Paulo City Council hosted the Casa Hunter conference in honor of World Rare Disease Day. The event provided a rich space for learning, exchanging information, and raising awareness about rare diseases. Abranghe was present, promoting AEH to parliamentarians, representatives of public institutions, health professionals, and leaders of associations, with a focus on the discussion on access to medicines.
From Daphne Dumbrille, HAE Canada
To kick off 2025, HAE Canada Board members Michelle Cooper and Kim Speiss appreciated the opportunity to escape the Canadian winter to attend the American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO)’s Joint Congress in San Diego, California, from February 28th to March 3rd. They were pleased to observe numerous posters related to HAE while proudly presenting the HAE Canada poster titled “Icatibant Use in Patients with HAE with normal C1-INH”. When they were not presenting the poster, they connected with leading HAE experts and attended presentations on various topics concerning HAE. On Saturday evening, they enjoyed meeting up with the other Canadian attendees at the Canadian Society of Allergy and Clinical Immunology’s Reception. Michelle and Kim left the conference feeling appreciative and encouraged by the dedication of numerous physicians and scientists to HAE research.
Each year, on February 28th, over 100 countries come together to celebrate Rare Disease Day. This day of unity raises awareness and encourages policy change for over 300 million people across the globe with a rare disease. To mark this occasion, HAE Canada organized for Winnipeg’s Esplanade Riel Bridge and the Winnipeg Sign to be one of many Canadian monuments to shine pink, purple, blue, and green to Light Up for Rare. It is incredibly impactful to witness monuments across the globe lit up in the Rare Colours to bring a spotlight to rare diseases. We applaud RareDiseaseDay.org, along with the Canadian Organization for Rare Disorders, for organizing the event.
As mentioned in the November 2024 issue, HAE Canada provided Canada’s Drug Agency (CDA), the health agency responsible for advising the government on treatment reimbursement, with a patient submission to advocate for access to icatibant for Canadians with HAE with normal C1. We are pleased to announce that after reviewing HAE Canada’s submission, along with contributions from other sources such as the Canadian HAE Network (CHAEN) and assessing the evidence demonstrating the efficacy of icatibant for this patient population, the CDA has concluded that individuals with HAE with normal C1 should have access to icatibant. Although this decision does not immediately grant access or reimbursement to people with HAE with normal C1, it represents a significant milestone as it is the first successful step towards achieving access. We will now focus on advocating for each province and territory to include normal C1 patients in their respective formularies.
More patients in Canada now have access to Orladeyo, the first oral preventative treatment for HAE. We are thrilled to report that people with Type 1 or 2 HAE in Ontario now have the option to access and be reimbursed for Orladeyo. Ontario is the fifth province to add Orladeyo to the provincial formulary, behind Alberta, British Columbia, Nova Scotia, and Quebec. Based on the speed of negotiations between provinces and the manufacturer, BioCryst, we are confident that it will not be long before the rest of Canada will have access to Orladeyo.
HAE Canada Board and staff are looking forward to our next 2 projects. At the end of March, the Board will travel to Ottawa for our annual Board Retreat to determine our next projects and initiatives to ensure we fulfill our mission and vision set out in our current Strategic Plan. We are also looking forward to hosting our next hybrid Patient Information Update from Timmins, Ontario, in early April.
We are pleased to see a growth in our membership, which reinforces our dedication to our mission and values. This steady increase motivates us to remain committed to assisting HAE patients in achieving the quality of life they deserve.
Country update from HAEi’s Javier Santana, Regional Patient Advocate, Central America & Caribbean
On 16 May 2025, our Costa Rican Member Organization representative, Tayra Corrales, will officially announce her book “Esta también soy yo” (This Is Me Too) – A life story as a hereditary angioedema patient.
“This book tells my story as a patient with a disease classified as rare. I tell you about my experience, everything I have learned over time, how I have done it and the tools that have worked in my life to be happier and more fulfilled every day, working from self-love and understanding of life. In addition, I want to expand knowledge of this disease and help more patients be diagnosed more efficiently and responsibly,” says Tayra.
The book will be available on the Amazon Store starting 16 May.
From Rosa María Pérez González, HAE Cuba
Hello, greetings from Cuba to the entire HAE Community worldwide.
It is always a pleasure and timely opportunity to express ourselves in the editions of this Magazine.
The year 2025 started with great momentum in our nascent association, so much so that many of us who are part of it and are located in different provinces of the country have managed to join and interact using social media through a WhatsApp group. We are 23 members so far, including patients, caregivers, and physicians, through which we monitor crises, show solidarity and support, and, most importantly, empathize with one another. Most importantly, we know we are not alone.
Our country, unfortunately, continues to lack modern therapies of choice for the treatment of crises, much less for prevention, and this negatively impacts the quality of life of each and every one of us. Currently, the therapeutic arsenal we have access to, albeit with restrictions, consists of danazol, transexamic acid, and fresh frozen plasma for the most severe attacks, so we experience the symptoms more acutely, and the stress it generates is very high.
For this reason, and given the need even to access diagnosis for a group of families with clinical symptoms of the disease, we are committed to making ourselves visible to Cuban Public Health authorities, who, despite facing significant financial limitations, are unaware of the risks posed by this disease, nor are they prepared at the different levels of health care to address it.
Likewise, we have begun a process of interaction with medical specialists and, through them, with the Cuban Society of Immunology to achieve collaborative work with patients and thereby activate care mechanisms and resource allocation to ensure the availability of specific therapies in the country. On May 16, we will celebrate International HAE Day as part of the international patient community, using this day to raise greater awareness about this rare disease in our country.
News from East Asia
Country updates shared by Yong Hao Lim, HAEi’s Regional Patient Advocate, East Asia
Good news from East Asia: Clinical trial participation is increasing across the region. Japan, Korea, Hong Kong, and Singapore have all made progress, with Singapore participating as a trial site for the first time. China is also advancing, with plans for a trial on domestically produced plasma-derived treatments and the registration of a new trial evaluating a siRNA therapy for HAE. These developments mark an important step toward expanding treatment options for patients.
Treatment availability is also improving. Japan recently introduced a new prophylactic treatment option, and we remain hopeful that access will continue to expand throughout the region.
From Peter Strahl, HAE Hungary
Rare Day 2025 – Budapest Semmelweis University February 22, 2025
The aim of Hungarian Rare Day is to increase awareness of rare diseases among decision-makers, healthcare professionals, and society. Information is the key to improving the living conditions of people with rare diseases.
Raising awareness is, therefore, one of our primary goals, because:
- Action taken simultaneously in many places and countries can ensure that the voices of rare patients are heard by as many people as possible.
- A day focused on rare diseases can bring hope and information to people living with rare diseases, their caregivers, and families.
- We want European rare patients to have equal access to care and treatment.
- We need action that unites all stakeholders in rare diseases for common goals.
- Research and the care system require greater financial support, and we need more research and greater efforts for the benefit of rare patients.
- We need coordinated political action programs at national and international levels.
On the occasion of Rare Disease Week and World Day, the Association of People with Rare and Congenital Disorders (RIROSZ), with its 61 member organizations, including the Hungarian HAE, organized the “Rare Day” for the 18th time this year on Saturday, February 22, 2025, in the Semmelweis University building in Budapest.
The program aims to create a forum where the relevant interest groups can jointly discuss the situation and plans of people living with rare diseases.
Several of our fellow patients represented our organization at the event. Among our interested parties were other patients suffering from rare diseases, pharmaceutical company representatives, health consultants, and family doctors. We tried to give a comprehensive picture of what surrounds us in relation to HAE from both the patient’s and doctor’s perspectives.
We agreed with other colleagues suffering from rare diseases and representatives of patient organizations that involving our fellow patients in daily work, and raising awareness of other fellow patients who may need help and more information, are the most difficult tasks at the moment.
From Priyanka Meduthuri, HAE India
HAE India at 2025 HAEi Regional Conference APAC: Strengthening Our Community
The 2025 HAEi Regional Conference APAC in Manila, Philippines (March 7-9) was an inspiring and insightful event for the HAE India community. Five Indian patient families attended, gaining valuable knowledge on improving diagnosis, treatment access, and patient support.
Dr. Ankur highlighted how family screening and proper diagnosis can help identify more HAE patients in India. HAE India’s work was showcased through a recorded introduction by President Pravalika Meduthuri, strengthening our presence on the global platform.
Our youngsters, Akanksha and Shashwath, had an engaging experience in the youth program, with Akanksha also participating in the HAEi LEAP program. We connected with advocates from Asia and Australia, learning best practices to implement in India.
A key highlight was discussions with pharmaceutical companies, providing insights into the future of HAE treatment and access.
This conference reinforced our commitment to raising awareness, improving access, and supporting the Indian HAE community. We return with inspiration and a strong action plan for the future.
News from Parichehr Bahraini, HAE Iran
The “Understanding HAE” patient guide is now available in Persian.
The Immunology, Asthma and Allergy Institute (IAARI), Tehran University of Medical Sciences, is proud to announce that the translation of the “Understanding HAE” patient guide has been completed in collaboration with HAEi.
This significant collaboration aims to enhance accessibility to critical knowledge and resources within the community.
This partnership not only strengthens the resources available for HAE patients in Iran but also reflects our commitment to global cooperation and knowledge sharing. By translating this essential guide, we aim to empower patients and their families with the knowledge they need to manage their health effectively.
The translated version of the patient guide will be officially available for download on the IAARI’s website and the Immunodeficiency Patient Advocacy (IDPA) website, as well as on haei.org.
We encourage community members, healthcare professionals, and advocates to share this resource widely, ensuring that those in need receive the information necessary for better health outcomes.
IAARI’s commitment to fostering understanding and collaboration across nationalities is reflected in this project. The work involved a meticulous process of translation and editing, ensuring that the essence of the original content was preserved while adapting it to meet the needs of Iranian readers.
We hope to continue our activities to provide updated information for Iranian HAE patients.
From Bettina Carty, HAE Ireland
HAE Ireland Patient Day 29th March 2025
On 29th March, 2025, HAE Ireland had its first official patient meeting – a highly successful event with patients traveling from all parts of the country to St. James’s Hospital in Dublin, who had kindly offered us their seminar room and support for the occasion.The event started with Bettina Carty, national contact for HAEi, and Yujin Yamamoto, involved with HAE Japan, currently based in Ireland, providing fantastic support to HAE Ireland, welcoming the patients and speakers. Bettina discussed the importance of a group like HAE Ireland, the support and applications that are available through HAEi, traveling with the disease, and future events for HAE Ireland, including hae day :-) on 16th May. Yujin spoke on the availability of medications available to patients and newer treatments that have been developed.
Consultant Immunologist Professor Niall Conlon from St. James’s and Derval Reidy, Assistant Director of Nursing in St. James’s Clinical Research Facility, spoke on medications available in Ireland, legalities in getting new drugs for patients and modern drug trials, and how they work. They also discussed the importance of patient advocacy and how our voices can help us with these trials coming to Ireland, the availability of new treatments, and how they can help in the improvement of quality of life for HAE patients in Ireland.
4th ITACA National Congress and AAEE ASSEMBLY
The 4th ITACA National Congress concluded with the excellent success of the doctor-patient session dedicated to patients affected by the disease and organized in collaboration with our association.
Also broadcast in live streaming with excellent participation, the session gave patients the opportunity to interact directly with doctors. Our association, in close collaboration with the scientific society ITACAngioedema , has always been committed to spreading knowledge of the disease and supporting patients with the aim of increasingly improving their quality of life.
Source: angioedemaereditario.org, 31 March 2025
From Ganesh Dahal, HAE Nepal
HAE Nepal was successfully registered as a non-profit Organization in March 2025.
From Natasha Jovanovska Popovska, President, HAE North Macedonia
1. A representative of HAE Macedonia, Verche Jovanovska Jankovska, visited the President of the Republic of North Macedonia, Godana Siljanovska Davkova, on behalf of the National Rare Disease Alliance. Through an emotional conversation, Verche, alongside several other members of the National Rare Disease Alliance, discussed the challenges faced by rare disease patients in Macedonia with the President of North Macedonia.
The meeting began with the presentation of a handwritten letter from the brother of a patient with a rare disease and special needs, he had asked the President of North Macedonia to help his brother. The difficulties that rare disease patients face in their daily lives were discussed at the meeting, alongside the challenges in the social and educational systems, the stigmatization they face every day, and the inaccessibility of patients with special needs. The battles that these patients have won in North Macedonia and the battles that lie ahead were also discussed.
At the meeting, Verche Jovanovska Jankovska shared the story of a nine-year-old child with Hereditary Angioedema who once suffered a stomach attack, but after receiving the treatment, he managed to run a marathon and was declared the hero of the day. The President stated that it was unacceptable to discuss these battles only once a year on Rare Disease Day – February 28 – and suggested that patients with rare diseases should fight even more vehemently but also be united to achieve their goals and increase their visibility.
Possible solutions and approaches were also discussed, such as the National Rare Disease Strategy, which calls for systemic solutions for rare disease patients, guarantees of access to education, social services, employment opportunities, and decent work, which will certainly result in a discussion in Parliament. At the meeting, which lasted over 2 hours, the President stated that she would give the rare disease patients her unwavering support everywhere.
2. HAE Macedonia representative Jovan Jankovski, as a member of the National Rare Disease Alliance, attended a meeting initiated by the Rare Disease Committee, which marked a rare occasion for patient representatives to be invited to such a meeting.
The meeting was to determine guidelines and directions for the cooperation between the Rare Disease Committee and the National Rare Disease Alliance. The importance of improved and faster diagnosis of rare diseases was highlighted. It was agreed that a meeting would be held in the period from 28-30.04.2025 to adopt a rare disease strategy.
The strategy would include 3 segments: Health (Ministry of Health and the Rare Disease Committee), education, and social segment.
Additionally, there was talk about unifying and accelerating the procedures for receiving therapy from hospitals.3. HAE Macedonia representative Natasha Jovanovska Popovska, on behalf of the National Rare Disease Alliance of North Macedonia, as well as Aleksandar Petrovski, also from the National Alliance, met Žaklina Peševska, an MP of the Republic of North Macedonia and President of the Equal Opportunities Committee, as well as Lidija Petkoska, an MP at the Assembly of the Republic of Macedonia.
The purpose of the meeting at the National Parliament was the initiative by the National Rare Disease Alliance to get the UN Resolution on Addressing the Challenges of People Living with Rare Diseases and Their Families ratified. The Resolution was adopted in December 2021, with a consensus of 193 member states. The meeting discussed the technical aspects and steps for ratification of this important document, which would become a foundation for adopting the National Rare Disease Strategy of the Republic of North Macedonia. In addition, the MPs expressed interest in joining the National Alliance of Rare Diseases of Macedonia (NARBM) campaign to raise awareness about rare diseases during the celebration of Rare Disease Day.
MPs Žaklina Peševska and Lidija Petkoska had the opportunity to learn about the challenges that patients with rare diseases face every day, but also about the successes of some of the NARBM representatives in fighting for appropriate therapy for the patients they advocate for, as well as achieving success internationally.
From HAE Panama
In February 2025, patients and members of the Panamanian Hereditary Angioedema Patient Association received the welcome news that we now have another drug registered in the country. In this case, the generic Icatibant.
In addition, some members of the patient association (photo 1) met with the representative of the drug’s pharmaceutical company. During this meeting, more details were learned about the drug.
On February 27, members of the board of directors and patients held a meeting at the Ministry of Health (MINSA) with the coordinator of the rare disease program and 2 other members of this program.
At this meeting, we had the valuable company of Mr. Javier Santana, our representative at HAEi. During the meeting, we learned about the status of the program and how it is working for HAE patients. Mr. Javier Santana was able to contribute ideas to improve drug procurement and benefit patients. We are incredibly grateful that he joined us at this important meeting.
At this moment, we are organizing activities for International HAE Day.
From HAE Poland
Celebrating 20 Years: HAE Poland’s Regional Conferences in 2025
The year 2025 marks a significant milestone for the Swelling Beautifully Association (also known as HAE Poland) as it celebrates the 20th anniversary of its official registration. However, HAE advocacy efforts in Poland began even earlier, 2 years before the organization was formally established. To commemorate this occasion, the Board of Directors opted for a series of 3 regional events instead of a single central gathering, ensuring greater accessibility for patients and broader participation.The first HAE Regional Conference took place on Saturday, 22 March 2025, in Warsaw, Poland, bringing together nearly 100 participants, including patients, caregivers, healthcare professionals, and industry partners. It was dedicated to patients from the Lubelskie, Łódzkie, Mazowieckie, Podlaskie, Świętokrzyskie, and Warmińsko-Mazurskie voivodeships.
The conference opened with a retrospective on 2 decades of advancements in HAE patient care in Poland, highlighting how advocacy has transformed patients’ lives. A key figure in this progress has been Michał Rutkowski, President of the Swelling Beautifully Association and Vice President of HAE International, whose leadership has played a pivotal role in securing access to and reimbursement for modern HAE treatments, including both on-demand therapies and long-term prophylaxis.
Several HAE expert physicians provided valuable updates:
- Dr Marcin Stobiecki, from HAE National Center in Kraków, reviewed current treatment availability and reimbursement rules, helping patients navigate their options.
- Dr Marcin Kurowski, from HAE Regional Center in Łódź, touched on ongoing clinical trials for potential new medications, emphasizing the importance of patient participation in research.
- Dr Tomasz Matuszewski, from HAE Regional Center in Warsaw, provided a brief update on diagnosis and treatment strategies for children and pregnant women, the most vulnerable patient groups.
Beyond medical advancements, the conference focused on strengthening patient connections. A dedicated storytelling session allowed patients to share personal experiences, creating an emotional and empowering space. These testimonials underscored the transformational impact of modern treatments, showing how individuals once severely affected by HAE can now lead a life comparable to those of healthy individuals thanks to improved access to medication.
Dominika Słodka, the Swelling Beautifully Association’s Regional Patient Advocate and leader of HAE Poland Youngsters’ Community, shared her journey as a member of the HAEi Youngsters Advisory Group. She emphasized the power of patient engagement and introduced youth-led initiatives, including the HAE Poland Youngsters Summer Workshops, which have already seen 2 successful editions.
Additionally, attendees had the chance to learn self-administration techniques through a dedicated training course available throughout the event.
The conference also introduced exciting resources from HAE International. Michał Rutkowski presented HAE TrackR, an extremely useful app that sparked great interest among participants, demonstrating its potential to help patients better track and manage their condition.
The event successfully combined education, advocacy, and community building, reinforcing the Swelling Beautifully Association’s dedication to improving the lives of HAE patients in Poland.
To relive the highlights of the 2025 HAE Poland Regional Conference, please watch the video below the images.
From HAE Puerto Rico
In Puerto Rico, CSL Behring has started efforts to distribute its medications on the island and provide physicians with information about them. The company recently opened its fourth plasma collection center on the island and held a facility event to share more information with physicians. Puerto Rican patients remain engaged and attentive to any changes regarding HAE on the island. Many pharmaceutical companies held events at the start of 2025 regarding new HAE research and future developments, and Puerto Rican HAE patients attended and participated in these.
From Chairman Elena Bezbozhnaya, HAE Russia:
On November 23, a webinar, “Treatment of HAE and a legal hour”, was held, organized by the Interregional Public Organization “Society of Patients with Hereditary Angioedema”. Participants from various regions of Russia learned about methods of treating a rare disease from experts, including the head of immunopathology, Elena Latysheva, who presented an overview of modern drugs and new methods, such as gene therapy. Lawyer Ilya Ushankov spoke about changes in the Clinical Guidelines for the Treatment of HAE for 2024, including the new long-term prevention drug “Lanadelumab”. The webinar also included a survey of participants on the availability of therapy and answers to patients’ questions regarding treatment regimens and obtaining drugs in remote regions.
The “Patient Assistance Center” was opened and created by the team of the International Public Organization “OPNAO” with 10 years of experience working with people diagnosed with “hereditary angioedema”. The “Patient Assistance Center” is a continuation of the mission of the International Public Organization “OPNAO”. The organization offers patients free support, including individual routing, expert consultations, and legal and psychological assistance. Webinars and face-to-face events are regularly held to communicate and exchange experiences between patients. The main goal of the Center is to unite people with rare diseases, protect their rights, and help them lead a full life. The organization invites everyone who is ready to support this initiative to cooperate.
On February 15, 2025, the International Public Organization “OPNAO” held a webinar, “NAO and society: how to talk about your illness?” where the main speaker was clinical psychologist Ekaterina Shutkova. Participants shared their experiences and discussed the adaptation of people with hereditary angioedema (HAE) in society. The psychologist gave advice on communicating about the disease at work and at school, emphasizing the importance of open dialogue and preparing information about HAE. Strategies for overcoming the fear of the diagnosis and ways to help loved ones understand the peculiarities of life with HAE were also discussed. The webinar brought together patients from 20 regions of Russia, strengthening the community and sharing experiences. The IPO “OPNAO” plans to continue holding such events to support patients and their families.
At the meeting organized by the Circle of Good Foundation, together with the Commission of the Public Chamber of Russia on Social Partnership, Care and Development of Inclusive Practices, the Autonomous Non-Commercial Organization “Patient Assistance Center”, and the IPO “OPNAO”, the chairman of the board, Archpriest Alexander Tkachenko, noted that thanks to regulatory documents and cooperation with NGOs and healthcare, children with rare diseases can receive medications without delays. Elena Bezbozhnaya, Head of the Interregional Public Organization “Society of Patients with Hereditary Angioedema”, presented the results of a survey among patient organizations, which showed a successful transition to the adult healthcare sector in some regions. Lawyer Ilya Ushankov emphasized the importance of NGOs in supporting young patients when transitioning to the adult network. The participants of the round table discussed the mechanisms of a “seamless” transition and prepared proposals for the government to improve the rights and opportunities of patients 19+.
The IPO “OPNAO” held a webinar, “Legal Hour”, dedicated to the rights of patients with hereditary angioedema (HAE). Participants learned about their rights to quality medical care and ways to protect these rights when interacting with medical institutions. Support resources for patients with HAE were also presented. During the webinar, the lawyer answered questions from participants regarding the legal aspects of the disease. The event took place on March 15, 2025, and brought together representatives of patient organizations and parents of patients.
A psychological training event, “Feel the support in yourself”, was held on March 29, 2025 from 1:30 PM to 3:00 PM in an online format. At the event, patients learned how to accept the diagnosis and adapt to new living conditions. The training will help them understand normal psychological reactions to the disease, master techniques for managing negative emotions, and build supportive relationships with themselves and others. Speaker – medical psychologist of the Dmitry Rogachev National Medical Research Center for Pediatric Hematology and Immunology, E.S. Shutkova.
From Sarah Smith, President, HAE Spain (AEDAF)
AEDAF: 27th General Assembly & Annual Meeting
The 27th General Assembly and Annual Meeting of AEDAF took place on Saturday, 22 February, in Vall d’Hebron Hospital in Barcelona. This is the first time that we have held our Annual Meeting outside Madrid, in line with our decision to start alternating our Annual Meetings between the 3 CSURs (Centers, Services and Units of Reference) for Hereditary Angioedema in Spain (La Paz Hospital in Madrid, Vall d’Hebron Hospital in Barcelona and Virgen del Rocio Hospital in Seville), in an attempt to make it easier for more of our members around the country to attend these meetings.After the General Assembly, we had a coffee break in the hall of the Children’s Hospital, where all the attendees had the opportunity to visit the Travelling Exhibition “Putting a Face on HAE” (created by Biocryst with the collaboration of AEDAF). We then had a very interesting, informative Annual Meeting, which can be seen in its entirety on our YouTube channel.
hae day :-) 2025
To commemorate hae day :-) 2025, in addition to encouraging our members to take part in HAEi’s global activity campaign, #active4HAE, AEDAF is organizing a benefit concert that will take place on Sunday, March 25 at 12 noon in the Teatro Monumental of Madrid.Website and HAEi Connect
AEDAF now has a new, updated website with a more modern, fresher look. The new version was launched on 19 February 2025.
On 24 January 2025, AEDAF also successfully completed the migration to the new HAEi Connect system.
From Ernst Greber, HAE Switzerland:
On Saturday, May 24, 2025, we will hold our annual patient meeting.
After a welcome coffee, various topics will be discussed.
- Various treatment options: Patients and HAE experts in conversation – valuable insights, information, and practical tips for everyday life.
- Healthy nutrition: The basis for a strong immune system and a better quality of life at any age.
- International HAE Association: Worldwide activities and goals.
After lunch, we will enjoy a tour of Zurich’s main train station or a walk around Zurich.
Following this, we will conclude the day with a final aperitif.
There is good news from the pharmaceutical industry, too: Swissmedic has granted CSL Behring approval for Andembry® (garadacimab) to prevent recurrent attacks of HAE.
From Marijk Beekman-Kortekaas, President, HAE the Netherlands
Can you believe the first quarter of the year has already flown by? Time sure does fly when you’re advocating and raising awareness. HAE the Netherlands has been very active these past few months, and we’re excited to share some of our recent activities with you. From engaging events to impactful initiatives, we’ve been working tirelessly to make a difference in the lives of HAE patients.
HAEi booklets
We are thrilled to announce that the Dutch translations of “Understanding HAE” and “Women with HAE” have been published on the HAEi website. We are grateful to HAEi for providing these valuable booklets, which have helped to increase awareness and understanding of HAE. Our role was to facilitate the translations, ensuring they are accessible to Dutch-speaking communities.In addition to the Dutch versions, we also took care of the Flemish translations for both booklets, ensuring that our Flemish-speaking friends and families can benefit from this important information as well.
So, what are you waiting for? Get over to the website and download the booklets!
Rare Disease Day
We were excited to participate in the national event for Rare Disease Day in the Netherlands, organized by the National Alliance for Rare Diseases. The afternoon program featured presentations on European initiatives in the fields of healthcare and research, as well as European reference networks.During a pleasant buffet, there was plenty of time for networking with other patient organizations for rare diseases. The evening was dedicated to awarding patient advocates, researchers, and media personalities who have made significant contributions to improving the lives of those living with rare diseases.
Additionally, the palace in Baarn was illuminated as part of the #LightUpForRare campaign, showing solidarity and raising awareness for rare diseases.
Foto: Johan de Graaf
Collaborating for Better Care: Our Commitment to Rare Diseases
Along with other patient organizations dedicated to rare diseases, we’re actively working to improve Dutch policies related to rare diseases. Recently, we have been focusing on the process of designating Centers of Expertise for rare diseases. We are doing this through a discussion platform, where our organization plays a leading role. We lead the working group responsible for evaluating the forms that patient organizations use for assessment. Our objective is that these forms need to reflect what matters most to patients.Additionally, we are also engaged in shaping policies related to the organization of care for rare diseases. Collaborating with other patient organizations is crucial when striving to change policy, and it’s also a pleasure to work together with these dedicated groups. This collaboration ensures that our voices are heard, and it strengthens our collective efforts.
If the organization of treatment for rare diseases in the Netherlands is not well-organized, then the organization of treatment for HAE patients will also not be well-organized. Together, we are committed to making a difference.
Learning and Sharing: A Wonderful Day with AEDAF in Barcelona
We are pleased to share that our president recently attended the patient meeting organized by the Spanish association AEDAF in Barcelona. She was there to see how other member organizations conduct their patient meetings, as this is incredibly educational.Our president also loves chatting with HAE patients from other countries. We all face many of the same challenges, and it’s enlightening to learn how people in other countries handle them.
It was a fantastic day filled with so many interesting presentations. Despite her limited Spanish, our president had wonderful conversations with Spanish HAE patients. She left the meeting filled with great ideas that she plans to implement in the Netherlands.
We want to compliment the Spanish association AEDAF for the well-organized day and extend our heartfelt thanks to Sarah and Maria for their hospitality.
From Shradha Singhania, Patients Lead, HAE UAE
Recently, on 9th February 2025, an HAEi patient workshop in the UAE was organized in Abudhabi under the guidance of Michael Rutkowski.
Dr Marylin Karam was the guest speaker, and patients and caregivers were given opportunities to participate and share their experiences. It was a very informative and successful event hosted in Abudhabi.
From Angela Metcalfe, HAE UK
This year HAE UK were delighted to mark Rare Disease Day on 28th February, with several events held at the Houses of Parliament in London. These events were supported by Jim Shannon MP, and included a meeting of clinicians and patients, with a panel discussion hearing about individuals’ journeys with diagnosis and treatment pathways – 2 very different stories from HAE UK Trustees Rose Joseph and Dana Shapiro.
Leading UK Immunologist Dr Sorena Kiani and Consultant Nurse Christine Symons were the experts on the panel, explaining how important a good relationship is between patient and doctor to optimise the best treatment.
Many Politicians and Members of the House of Lords attended to hear the concerns, issues, and successes discussed – it was a great way to raise awareness of Hereditary Angioedema, and we received lots of interest in learning more about the condition.
From HAE Ukraine
This year, we continue the active work of the association, aimed at raising awareness of HAE in Ukraine. We are developing useful adapted materials for young patients and their parents, we involve families in participating in events, and we are preparing for a large-scale All-Ukrainian conference for the HAE day in May.
On February 28, on the day of the World Day of Rare Diseases, the “Ukrainian Patients Association for HAE” together with the public organization “Rare Immune Diseases” held the forum “Side by Side. Together we are strong!” dedicated to the support of patients with primary immunodeficiencies (PID) and hereditary angioedema (HAE).
Immunologists, allergists, nurses, patients, and their families gathered under the auspices of “Kyiv Nephrology Center” and “Children’s Hospital No.1” to discuss the most pressing issues of diagnosis, treatment, and support of patients by the medical community and state programs.
The forum program included speeches by leading specialists and managers of specialized hospitals, stories of projects and initiatives, master classes by nurses, and an “Ask the Doctor” session, where participants could get answers to their questions. Competitions with prizes were organized by animators for children.
Many thanks to our sponsors LLC “Takeda”, “Biopharma”, “Octapharma”, and the technical organizer “Open Likar”.
The event was an excellent platform for sharing experiences and creating new initiatives to support patients. Together, we are a force!
From Jess Meyers, Communications and Advocacy Manager, US HAEA:
Bringing Together the US HAEA Community at the 2025 National Summit
The HAEA is excited to announce the 2025 HAEA National Summit in Baltimore, Maryland, this summer from July 10-13.
The theme of this year’s Summit, “Embracing a Brighter Future,” reflects our community’s optimistic spirit and commitment to growth, connection, and empowerment. This unique gathering will bring together over 1,200 people with HAE, families, caregivers, healthcare professionals, and pharmaceutical company representatives for an unforgettable experience.
HAEA Grassroots Advocacy Leaders Amplify Our Collective Voice During Rare Disease Week
HAEA Grassroots Advocacy leaders celebrated Rare Disease Week by participating in the Everylife RDLA conference that took place in Washington, DC. Showcasing the resolve and persistence of the HAE community, our 18 HAEA Grassroots Advocacy Leaders from 14 different states met with over 55 legislative offices during the 2025 Rare Disease Week. Many legislators graciously met with HAEA advocates in person, including Congresswoman Emilia Sykes (D-OH), Congressman Max Miller (R-OH), Congressman Chuck Fleishman (R-TN), Congressman Paul Tonko (D-NY), Senator Marsha Blackburn (R-TN), Senator Eric Schmitt (R-MO), and Senator Cory Booker (D-NJ). With over 1000 rare disease advocates attending this event, we are positive that our voices were heard and made an impact on key decision-makers this year.
To learn more about the US HAEA Advocacy program, you can:
- Join the US HAEA Grassroots Advocacy network,
- Visit the HAEA website,
- Watch the 2025 Welcome to Congress and Legislative Update.
Celebrating hae day :-)
hae day :-), recognized every May 16th, is a powerful reminder of what we can achieve when we come together as a community. Established through Senate Resolution 286, championed by Senator Daniel K. Inouye, this day has grown into a global movement dedicated to raising awareness, advancing education, and advocating for improved treatments for HAE.
As we approach hae day :-), the US Hereditary Angioedema Association (HAEA) reaffirms our commitment to progress—ensuring that individuals with HAE continue to receive the support, education, and medical advancements they deserve.
This year, we will be mailing out a special hae day :-) poster to US HAEA members and encourage them to spread awareness by:
- Snapping a photo with their poster,
- Tagging the US HAEA when they post their photo to social media, and
- Joining Team USA for HAEi’s global #active4HAE campaign!
Advances in HAE Research
Understanding how HAE affects people’s lives is at the heart of our research efforts. By collecting data and publishing studies, we help doctors, patients, and policymakers see the full picture of living with HAE.
Recent US HAEA research efforts include:
Expert Consensus on HAE With Normal C1 Inhibitor
In a joint initiative with HAEi, the HAEA Research Team convened a distinguished international group of HAE experts to deliberate and reach a consensus on the latest science regarding genetic mutations, physiological markers, diagnosis, and treatment for HAE with normal C1 inhibitor. The research teams at HAEA and HAEi worked with the authors to finalize this landmark study, which was recently published in the prestigious medical journal – Clinical Reviews in Allergy & Immunology.Study of HAE Prevalence in the US
This study will provide the first comprehensive estimate of people with HAE (all types) in the US. The HAEA Research Team, in conjunction with HAE expert physicians and a team of demographers and statisticians, crafted a methodology for making this estimate using a large insurance claims database. Identifying the true prevalence of a rare disease, like HAE, enhances the development of targeted strategies for patient support, clinical awareness, and future drug development initiatives. The paper has been submitted to a prestigious medical journal, and we expect the published article to be available in the first half of 2025.The US HAE-C1INH-QoL Tool
In 2024, we completed work on a unique, groundbreaking study that seeks to measure how HAE affects overall quality of life. This US-validated HAE-specific tool overcomes the limitations of other currently available options and better reflects patients’ experience with HAE. In addition, our QoL instrument: (1) truly captures the way HAE affects the everyday life of individuals and families, and (2) will demonstrate (to health insurers and others) the value of life-changing improvements in health and QoL that result from modern HAE medicines.We published the results of our study in the prestigious Annals of Allergy, Asthma & Immunology in July 2024.
We completed a follow-up project to develop a condensed version of the instrument and establish a numerical value that indicates a meaningful QoL change when the instrument is administered over time. We will submit a manuscript to a peer-reviewed medical journal and expect the paper to be published by midyear 2025.
Pediatric Study
Preliminary work has established the scope and methodology for a study of how HAE affects children. The HAEA will partner with experts who have significant experience conducting research in the pediatric population. The work is scheduled to begin in early 2025, with plans to present preliminary findings at the 2025 US HAEA National Summit.Study of HAE’s Impact on Caregivers
The HAEA is dedicated to providing much-needed real-world evidence regarding the perspectives and burdens associated with caregiving for someone affected by HAE. We worked with health economists and HAE expert physicians to design a data-gathering survey administered in 2024. The data has been analyzed, and we are writing a manuscript that will be submitted for publication in the second quarter of 2025.
Clinical Trials Update
According to clinicaltrials.gov under the U.S. National Institutes of Health, the EU Clinical Trials Register, and the International Clinical Trials Registry Platform under the World Health Organization (WHO), the following trials are currently or soon to be recruiting (as of 21 March 2025).
Read more about these and other clinical trials at:
HAELO: a Phase 3 Study to Evaluate NTLA-2002 in Participants with Hereditary Angioedema (HAE)
United States
https://clinicaltrials.gov/study/NCT06634420A Study Of Navenibart in Participants with Hereditary Angioedema
United States
https://www.clinicaltrials.gov/study/NCT06842823A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Puerto Rico, Spain, Turkey, United Kingdom, United States,
https://clinicaltrials.gov/study/NCT05392114HAE Burden and Crisis Management
France – not yet recruiting
https://www.clinicaltrials.gov/study/NCT06806618Safety Study in Subjects ≥ 12 Years of Age with Hereditary Angioedema Switching to Garadacimab
No sites confirmed
https://www.clinicaltrials.gov/study/NCT06806657A Phase 2 In Adult Subjects with Hereditary Angioedema
United States, China, Germany – not yet recruiting
https://www.clinicaltrials.gov/study/NCT06846398Suicide Ideation in Hereditary Angioedema (HAE-SAFE)
No sites confirmed
https://www.clinicaltrials.gov/study/NCT06811467A Survey of Lanadelumab in Participants With Hereditary Angioedema
Japan
https://clinicaltrials.gov/study/NCT05397431A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema
United States, Canada
https://www.clinicaltrials.gov/study/NCT06007677PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial
Australia, Austria, Bulgaria, France, Germany, Greece, Israel, Japan, Netherlands, New Zealand, Spain, United Kingdom, United States
https://clinicaltrials.gov/study/NCT05511922Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Bulgaria, Canada, Czechia, France, Germany, Hungary, Israel, Poland, Spain, United States – enrolling by invitation
https://clinicaltrials.gov/study/NCT05396105Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema (RAPIDe-3)
United States, Puerto Rico
https://www.clinicaltrials.gov/study/NCT06343779Status of Dental Care Practices in Patients With Hereditary Angioedema
France
https://clinicaltrials.gov/study/NCT05776784Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C-1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
United States, Armenia, Bulgaria, Mexico, Peru, Romania, Serbia, Ukraine
https://www.clinicaltrials.gov/study/NCT06361537Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
Australia
https://clinicaltrials.gov/study/NCT05691361A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr) in Persons With HAE in China
China
https://clinicaltrials.gov/study/NCT06346899Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) with HAE Type I or II (KONFIDENT-KID)
United States
https://clinicaltrials.gov/study/NCT06467084Institutional Registry of Rare Diseases
Argentina
https://www.clinicaltrials.gov/study/NCT06573723Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE (CHAPTER-3)
United States, Hong Kong, Ireland, South Korea, United Kingdom -not yet recruiting
https://www.clinicaltrials.gov/study/NCT06669754Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with HAE (CHAPTER-4)
Hong Kong, United Kingdom – not yet recruiting
https://www.clinicaltrials.gov/study/NCT06679881Angioedema Biomarker Research Study
No sites confirmed
https://www.clinicaltrials.gov/study/NCT06210698Identification and Characterization of Genetic Variants in Hereditary Angioedema (GENOMAEH_01)
Spain – not yet recruiting
https://www.clinicaltrials.gov/study/NCT05833620
HAE-Related Scientific Publications
In addition to a wide range of case reports, reviews, and small case series, here are summaries of recently published HAE-related scientific papers. Data search was undertaken on 21 March 2025.
An evaluation of sebetralstat as the first oral on-demand therapy for hereditary angioedema
Henriette Farkas and Zsuzsanna Balla
The authors review sebetralstat, a promising option for rapid and effective on-demand treatment of HAE attacks. They conclude that oral sebetralstat provides a convenient alternative to injectable treatments, allowing patients to take the drug at the first sign of an attack, helping to resolve them quickly. They believe its chemical properties indicate it has potential for reliable control of HAE attacks.
(Expert Opinion on Pharmacotherapy, 20 March 2025
Impact of dental procedures on hereditary angioedema attacks: An exploratory observational study
Valentin Nadasan, et al
The authors evaluate whether patients with HAE receive appropriate treatment following an HAE attack associated with a dental procedure. In diagnosed patients, adequate HAE-specific medications were mostly used. However, a proportion of patients refrained from dental treatment or were denied treatment by professionals, due to fears of HAE attacks.
(Oral Health and Preventive Dentistry, 14 March 2025)
Hereditary angioedema with normal C1 inhibitor: An updated international consensus paper on diagnosis, pathophysiology and treatment
Bruce L Zuraw, et al
The authors propose recommendations for the management of HAE with normal C1-inhibitor (HAE-nC1INH). They suggest their review and expert opinion on best practice will support physicians to better manage patients with HAE-nC1INH.
(Clinical Reviews in Allergy and Immunology, 7 March 2025)
HAE International (HAEi) and the US Hereditary Angioedema Association (HAEA) supported this important work on HAE with normal C1 inhibitor, and we’ll bring you the inside scoop on this project and publication in the next edition of Global Perspectives.
Hereditary angioedema (HAE) in China: Advancing awareness, access, advocacy and alliances from the greater bay area to the global HAE community
Philip H Li, et al
The authors provided an overview of the progress made in managing HAE in China. By bridging the gap between east and west, a Greater Bay Area HAE Alliance is providing optimal patient care. The authors conclude that uniting as a worldwide HAE community will significantly advance efforts to improve care for those affected by HAE.
(Clinical and Experimental Allergy, 4 March 2025)
Hereditary angioedema with normal C1 inhibitor: A quarter century of forward progress and persisting obstacles
Sandra C Christiansen, et al
The authors attempt to bring the numerous scientific developments over the past 25 years into a proposed classification scheme to help decision-making when doctors evaluate patients with recurrent angioedema. The authors hope that this approach will lay the groundwork for future advances in our understanding of HAE-nl-C1INH while bringing patients ever closer to the goal of leading a normal life.
(Journal of Allergy and Clinical Immunology – In Practice, in press 6 March)
Interplay between on-demand treatment trials for hereditary angioedema and treatment guidelines
Danny M Cohn, et al
The authors note that over the last 20 years, guidelines for HAE have undergone substantial revision. The paper reviews the evolution of on-demand treatment guidelines, the trials and data that supported revisions, and the changes to trial designs necessary to comply with guidelines.
(Journal of Allergy and Clinical Immunology, March 2025)
Clinical response and corresponding blood transcriptome pathways before and after treatment of hereditary angioedema prodromes compared to active swelling attacks
Debajyoti Ghosh, et al
The authors investigated whether there was value in treating HAE type-1 patients during prodromal symptoms. They conclude from their research that in patients who have a well-defined prodrome that historically leads to an attack, treatment at the prodrome stage is justified.
(J Allergy Clin Immunol, March 2025)
A COSMIN systematic review of instruments for evaluating health-related quality of life in people with hereditary angioedema
Irene Baroni, et al
The authors looked at the tools currently used to evaluate quality of life in people with HAE. They conclude that two, HAE-QoL and AE-QoL have evidence to support their use, but they believe more data is needed on their suitability amongst younger patients and disease specific elements in them. They believe doing so will improve the accuracy of patient-reported outcome measures in HAE.
(Health and Quality of Life Outcomes, February 2025)
Correction: Uncovering a novel SERPING1 pathogenic variant: Insights into the aggregation of C1-INH in hereditary angioedema
Lingxi Jiang, et al
The authors corrected an earlier paper that suggested they had identified a new genetic variant in HAE. They note that this variant was described by experts in 2022. (The article being corrected was published in September 2024.)
(Orphanet Journal of Rare Diseases, February 2025)
Indirect treatment comparison of lanadelumab and a C1-esterase inhibitor in pediatric patients with hereditary angioedema
Maureen Watt, et al
The authors set out to compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis treatments in children with HAE. Reviewing two papers that indirectly compared treatment, the authors found a trend toward greater efficacy and fewer adverse events in patients treated with lanadelumab every two weeks. They suggest further studies of larger groups of patients could assess this further.
(Journal of Comparative Effectiveness Research, February 2025)
Real-world effectiveness of lanadelumab in hereditary angioedema: Multicountry INTEGRATED observational study
Markus Magerl, et al
The authors aimed to find out the effectiveness of lanadelumab on attack-free rate. They did this by looking back at the medical records of patients with HAE who were treated with this medicine. The authors conclude that long-term prophylaxis with lanadelumab was effective in improving the attack-free rate in patients with HAE, and that effectiveness was rapid, starting from the first month of beginning treatment.
(Journal of Allergy and Clinical Immunology: In Practice, February 2025)
Worldwide prevalence of hereditary angioedema: A systematic review and meta-analysis
Samuel A Fisch, et al
The authors indicate that the true prevalence of HAE is uncertain. Through their research, they indicate that the available studies suggest the global prevalence of HAE is 1-2 individuals per 100,000 people. They believe a true estimate of prevalence will inform care for HAE.
(International Archives of Allergy and Immunology, January 2025)
New drugs for the treatment of hereditary angioedema
Giulia Costanzo, et al
The authors wanted to review existing and future treatment options for HAE. It is their expert opinion that innovative therapies may allow individualized action plans and reduce the complexity of treatment. They conclude that long-term prophylaxis with longer administration intervals and oral on-demand therapies will have a key role in the future.
(Expert Opinion in Biological Therapy, January 2025)
Efficacy and safety of donidalorsen in hereditary angioedema with C1 inhibitor deficiency: A systematic review and a meta analysis
Adarsh Raja, et al
The authors consider data currently available on a potential new treatment for HAE, called donidalorsen. They suggest that it significantly reduced the frequency of attacks, with dosing every four weeks being superior to every eight weeks. They also suggest the rate of side effects was the same for people treated with donidalorsen and people treated with placebo. The authors conclude that more frequent dosing may optimize treatment outcomes with donidalorsen in HAE.
(Archives of Dermatological Research, December 2024)
Advances in the pathogenesis of hereditary angioedema
Xiang-Yi Cui and Yu-Xiang Zhi
This review summarizes the recent progress in classifying and understanding the underlying processes that cause HAE, and the role this may have in leading to better diagnosis and treatment of the disease.
(Zhongguo Yi Xue Ke Xue Yuan Xue Bao, December 2024)
Hereditary angioedema in children: Review and practical perspective for clinical management
A Pagnier, et al
The authors review all aspects of how HAE is diagnosed and managed in children. They comment that lanadelumab (given subcutaneously) and berotralstat (given orally) have made long-term prophylaxis more usable in children with HAE. They conclude that management of HAE is particularly challenging in children and requires multiple stakeholders.
(Pediatric Allergy and Immunology, December 2024)
News from the Industry
13 January, 2025
Astria Therapeutics announces design of ALPHA-ORBIT pivotal Phase 3 trial of navenibart in HAE
Astra Therapeutics has announced its planned design of the ALPHA-ORBIT Phase 3 clinical trial of navenibart in people with HAE, which will include both every 3- (Q3M) and every 6-month (Q6M) treatment arms with the primary analysis at 6 months.
Global start-up activities are underway, and ALPHA-ORBIT is expected to initiate in Q1 2025, with top-line results anticipated in early 2027.
Jill C Milne, PhD, Chief Executive Officer at Astria, said: “We are thrilled to announce our planned Phase 3 design, which reflects feedback from regulators and is intended to support global registration for both Q3M and Q6M administration. With navenibart, we are pioneering patient-centric dosing flexibility in HAE with the goal of maximizing attack rate reduction with a compellingly low burden of treatment.”
Christopher Morabito, MD, Chief Medical Officer at Astria, said: “Our Phase 3 program was designed in collaboration with the patient community and physicians, is based on input from global regulatory authorities, and addresses the importance of providing options to patients for a disease that’s highly variable. Phase 3 preparations are underway, with trial initiation on-track and expected for this quarter. We are driven by the goal of bringing a potentially life-changing therapy to patients with HAE.”
ALPHA-ORBIT is designed as a global, randomized, double-blind, placebo-controlled Phase 3 pivotal clinical trial to evaluate the efficacy and safety of navenibart over a 6-month treatment period in up to 145 patients with Type 1 or Type 2 HAE. Patients will be randomized to receive one of three navenibart dose arms: 1) an initial 600 mg dose and followed by 300 mg Q3M, 2) 600 mg Q6M, and 3) 600 mg Q3M, or placebo. The dose arms support the potential to provide patient-centric dosing flexibility to people with HAE. The primary endpoint is time-normalized monthly HAE attacks at 6 months, and a key secondary endpoint includes the proportion of participants who are attack-free at 6 months. After 6 months, patients may be eligible to enter a long-term extension trial, in which all patients will be treated with navenibart (open-label) and which will include an open-label, patient-centric flexible dosing period. The navenibart Phase 3 program will consist of the ALPHA-ORBIT Phase 3 trial and the long-term extension trial, which are designed to support registration globally. The Phase 3 program was designed with input from the European Medicines Agency and the Company’s end of Phase 2 meeting with the US Food and Drug Administration (FDA) held in December 2024.
(Source: Astria)
13 January, 2025
Pharvaris outlines 2025 strategic priorities
Berndt Modig, Chief Executive Officer of Pharvaris, said: “This year is paramount to Pharvaris as we continue clinical development of deucrictibant to help address unmet needs for those living with bradykinin- mediated angioedema. Pharvaris is committed to generating robust clinical data to build a compelling package supporting deucrictibant’s efficacy and safety profile. Our team is focused on the execution of two Phase 3 clinical studies in HAE, the expansion of our pipeline into AAE, and preparations for commercialization of deucrictibant pending regulatory submission and approval; we have significant resources in place to support these strategic investments and provide value for our shareholders.”
2025 strategic priorities
Long-term prophylaxis of HAE attacks
- Initiated CHAPTER-3, a global pivotal Phase 3 study, evaluating deucrictibant for the prophylactic treatment of HAE attacks; topline data anticipated second half of
- Prophylactic open-label extension study CHAPTER-4 on track to initiate in first quarter of 2
On-demand treatment of HAE attacks
- Topline data from RAPIDe-3, a global Phase 3 study evaluating deucrictibant for the treatment of HAE attacks, anticipated first quarter 2026.
- Phase 2/3 open-label extension, RAPIDe-2, of deucrictibant immediate-release capsule for the treatment of HAE attacks is ongoing. All participants from RAPIDe-2 Part A, as well as participants who have completed RAPIDe-3, have or will be offered to enter Part B, the open-label extension study of deucrictibant immediate-release capsule (20 mg), which is the dose being used in RAPIDe-3 and currently the intended commercial
Clinical development of deucrictibant in AAE-C1 INH
- Clinical development plans of deucrictibant in acquired angioedema due to C1-INH deficiency (AAE-C1INH) underway. Pharvaris intends to initiate a clinical study in 2025 pending feedback from regulators.
Business updates
- Expansion of Pharvaris team to support deucrictibant launch preparedness, as well as business growth and
- HAE treatment experience and burden of disease data presented at recent medical Data from the Adelphi Disease Specific Programme, a real-world cross-sectional survey of physicians and people living with HAE, were presented at the Spanish Society of Allergology and Clinical Immunology (SEAIC) International Symposium and at the BSI Clinical Immunology Network (BSI-CIPN) Conference.
(Source: Pharvaris)
13 January, 2025
Astria Therapeutics announces design of ALPHA-ORBIT pivotal Phase 3 trial of navenibart in HAE
Astra Therapeutics has announced its planned design of the ALPHA-ORBIT Phase 3 clinical trial of navenibart in people with HAE, which will include both every 3- (Q3M) and every 6-month (Q6M) treatment arms with the primary analysis at 6 months.
Global start-up activities are underway, and ALPHA-ORBIT is expected to initiate in Q1 2025, with top-line results anticipated in early 2027.
Jill C Milne, PhD, Chief Executive Officer at Astria, said: “We are thrilled to announce our planned Phase 3 design, which reflects feedback from regulators and is intended to support global registration for both Q3M and Q6M administration. With navenibart, we are pioneering patient-centric dosing flexibility in HAE with the goal of maximizing attack rate reduction with a compellingly low burden of treatment.”
Christopher Morabito, MD, Chief Medical Officer at Astria, said: “Our Phase 3 program was designed in collaboration with the patient community and physicians, is based on input from global regulatory authorities, and addresses the importance of providing options to patients for a disease that’s highly variable. Phase 3 preparations are underway, with trial initiation on-track and expected for this quarter. We are driven by the goal of bringing a potentially life-changing therapy to patients with HAE.”
ALPHA-ORBIT is designed as a global, randomized, double-blind, placebo-controlled Phase 3 pivotal clinical trial to evaluate the efficacy and safety of navenibart over a 6-month treatment period in up to 145 patients with Type 1 or Type 2 HAE. Patients will be randomized to receive one of three navenibart dose arms: 1) an initial 600 mg dose and followed by 300 mg Q3M, 2) 600 mg Q6M, and 3) 600 mg Q3M, or placebo. The dose arms support the potential to provide patient-centric dosing flexibility to people with HAE. The primary endpoint is time-normalized monthly HAE attacks at 6 months, and a key secondary endpoint includes the proportion of participants who are attack-free at 6 months. After 6 months, patients may be eligible to enter a long-term extension trial, in which all patients will be treated with navenibart (open-label) and which will include an open-label, patient-centric flexible dosing period. The navenibart Phase 3 program will consist of the ALPHA-ORBIT Phase 3 trial and the long-term extension trial, which are designed to support registration globally. The Phase 3 program was designed with input from the European Medicines Agency and the Company’s end of Phase 2 meeting with the US Food and Drug Administration (FDA) held in December 2024.
(Source: Astria)
21 January, 2025
Pharming to nominate biopharmaceutical leader Fabrice Chouraqui as new Executive Director and Chief Executive Officer
Pharming Group announced that the Board of Directors has nominated Fabrice Chouraqui to become Pharming’s new Executive Director and Chief Executive Officer, succeeding Sijmen de Vries.
Pharming will nominate Mr Chouraqui for the appointment as Executive Director and Chief Executive Officer for a term of four years at an upcoming Extraordinary General Meeting of Shareholders (EGM).
Upon the appointment of Mr. Chouraqui, Sijmen de Vries will resign from the Board of Directors. To ensure a smooth hand-over of tasks and responsibilities, Mr. de Vries will remain a strategic advisor to the new CEO until 31 December 2025.
Dr Richard Peters, Chairman of the Board of Directors, commented: “After an extensive search, the Board and I are confident that Fabrice Chouraqui is the right candidate to lead Pharming and continue to implement our growth strategy, shaping Pharming into the rare disease company of choice. Fabrice is a deeply experienced global pharmaceutical and biotechnology executive, who brings a wealth of profound global expertise and experience, across the entire biopharmaceutical value chain, to Pharming. On behalf of the entire Board of Directors, I would like to thank Sijmen de Vries for his great commitment to Pharming over the past 16 years and for creating the company that it is today, serving patients and paving the way for delivery on the company’s strategy for growth. We are grateful that Sijmen will continue to be available through the end of the year to ensure a smooth hand-over with Fabrice.”
Sijmen de Vries, Chief Executive Officer, commented: “I am very pleased with the nomination of Fabrice Chouraqui as my successor. Fabrice brings strong leadership experience with global pharmaceutical companies and highly innovative biotechnology companies to Pharming. To make way for Fabrice to take over the executive leadership of the Company, I will resign from the Board of Directors upon the appointment of Fabrice by our shareholders. I look forward to supporting Fabrice, working closely together through the remainder of the year, and ensuring a smooth hand-over.”
Fabrice Chouraqui commented: “I am honored to have the opportunity to take over the leadership of Pharming, building on the strong foundation and joining at a time when I can help propel the company through its next stage of growth in the rare disease space. I am impressed by Pharming’s growing portfolio of products and commercial opportunities which will pave the way for us to become the rare disease company of choice.”
(Source: Pharming)
21 January, 2025
KalVista announces orphan drug designation and nda submission for sebetralstat in Japan for hereditary angioedema
KalVista Pharmaceuticals announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted sebetralstat Orphan Drug Designation. The Company has also submitted a New Drug Application (NDA) for sebetralstat to the Agency. If approved, sebetralstat would be the first oral on-demand treatment for HAE in Japan.
Ben Palleiko, CEO of KalVista, said: “The submission of our NDA for sebetralstat in Japan represents another key step toward our efforts to make this important new treatment available to as many people living with HAE as possible. The Orphan Drug Designation not only acknowledges the critical need for new, effective treatments for HAE in Japan but underscores the potential of sebetralstat to provide meaningful relief for people who have faced ongoing challenges with existing options. We are proud to be at the forefront of advancing care for the HAE community.”
The NDA submission is supported by previously disclosed results, including data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open-label extension trial.
(Source: KalVista)
22 January, 2025
Intellia Therapeutics announces first patient dosed in the HAELO Phase 3 study of NTLA-2002, an investigational in vivo CRISPR gene editing treatment for hereditary angioedema
Intellia Therapeutics announced that the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of HAE. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the Company’s plans for a US launch in 2027.
Intellia President and Chief Executive Officer John Leonard MD, said: “We are pleased to have initiated dosing in the HAELO Phase 3 study as we are in our final lap of clinical development for NTLA-2002. With the promising data we’ve presented thus far, we believe patients could achieve independence from both HAE attacks and medications required to treat this disease. We look forward to presenting longer-term data from the ongoing Phase 1/2 study later this year highlighting the durability of effect of NTLA-2002.”
Dr Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research Inc, commented: “We are excited to have treated the first patient in the US with a new generation of therapy that could potentially provide patients with lifelong relief from the primary symptoms of HAE.”
(Source: Intellia)
30 January, 2025
Julie Kim will succeed Christophe Weber as CEO of Takeda in June 2026
Takeda announced that its Board of Directors made the decisions unanimously to appoint Julie Kim, currently President of Takeda’s US Business Unit, as the successor to Christope Weber, Takeda’s President, Chief Executive Officer (CEO) and Representative Director, when Mr Weber retires from the company in June 2026.
Mrs Kim will be proposed as a candidate for election to the Board at Takeda’s Annual General Shareholders Meeting held in June 2026. Mr Weber will not hold a Board seat after retiring from Takeda.
Christophe Weber commented: “For several years I have worked with the Board to ensure a smooth succession. The Board’s selection of Julie is outstanding. I have worked closely with Julie for the past six years and have witnessed first hand her values, intellect, grit and dedication to our people and patients.”
Julie Kim commented: “Takeda is a unique company, and I am deeply honored to have been chosen to lead it. Thanks to Christophe’s phenomenal leadership, Takeda has become a global biopharmaceutical powerhouse with a promising late-stage pipeline. I am excited to guide Takeda through the next phase, together with our exceptional and talented people.”
(Source: Takeda)
3 February, 2025
Astria Therapeutics to Present at Upcoming Western Society of Allergy, Asthma & Immunology Annual Scientific Session
Astra Therapeutics has announced today announced that it will present quality of life results from the Phase 1b/2 trial of navenibart (STAR-0215) in a poster displayed at the Western Society of Allergy, Asthma & Immunology (WSAAI) Annual Scientific Session in Waimea, Hawaii from Sunday, 9 February through Thursday 13 February 2025.
Dr Marc A Riedl, Professor of Medicine and Clinical Director of the US HAEA Angioedema Center at the University of California, San Diego, will present quality of life data from the ALPHA-STAR trial of navenibart in an encore presentation of a poster titled: “Navenibart (STAR-0215) Induces Rapid Improvements of Quality of Life in HAE Patients in the ALPHA-STAR Trial.”
The poster will be exhibited from 3:00pm ET on 9 February until the event’s conclusion on the evening of 13 February 2025.
(Source: Astria)
10 February, 2025
KalVista Pharmaceuticals presents new sebetralstat data at the Western Society of Allergy, Asthma & Immunology 2025 Annual Meeting
KalVista Pharmaceuticals announced the presentation of novel data related to long-term prophylaxis and sebetralstat at the Western Society of Allergy, Asthma & Immunology (WSAAI) 2025 Annual Meeting taking place in Hawaii from 9-13 February 2025.
Raffi Tachdjian MD MPH, Associate Clinical Professor of Medicine and Pediatrics in the Division of Allergy and Clinical Immunology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA), presented data on the Impact of Long-Term Prophylaxis (LTP) Adherence in Hereditary Angioedema Patients: Results of a Claims Database Analysis.
- According to a US commercial claims analysis, nearly 40% of HAE patients who initiated LTP had substantial refill gaps in claims over 12 months, with more than half of those discontinuing LTP.
- For patients with substantial refill gaps, which increase the likelihood of non-adherence, on-demand claims remained unchanged before and after one year of starting LTP.
Marc A Riedl MD, Professor of Medicine and Clinical Director, US Hereditary Angioedema Association Center at the University of California, San Diego, presented data on the Safety and Effectiveness of Sebetralstat in Patients with Hereditary Angioedema Receiving Long-Term Prophylaxis: Interim Analysis from the KONFIDENT-S Open-Label Study.
- Participants receiving LTP treated 382 attacks with sebetralstat (mean 1.7 attacks per month), of which more than half involved the abdomen and/or larynx.
- Sebetralstat enabled early treatment (median 6 minutes) and early symptom relief (median 1.3 hours), and was well-tolerated, regardless of LTP mechanism of action or route of administration.
Michael E Manning MD, Allergist-Immunologist at Allergy, Asthma and Immunology Associates, Ltd, Scottsdale, Arizona and Past President of WSAAI, presented Sebetralstat for the Treatment of HAE Attacks in Patients Receiving Berotralstat: Interim Analysis from the KONFIDENT-S Open-Label Study.
- Participants receiving berotralstat treated 178 attacks with sebetralstat (mean 1.8 attacks per month).
- Sebetralstat enabled early treatment (median 20 minutes), early symptom relief (median 1.3 hours), and was well-tolerated with no increase in gastrointestinal side effects.
(Source: KalVista)
10 February, 2025
BioCryst presents new Orladeyo (berotralstat) results from APeX-P pediatric trial at 2025 American Academy of Allergy, Asthma & Immunology / World Allergy Organization Joint Congress
BioCryst Pharmaceuticals announced that the company will present five abstracts featuring new clinical and real-world outcomes with oral, once-daily Orladeyo (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) joint congress.
Included among these is a late-breaking abstract that highlights the first presentation of results from the APeX-P trial evaluating oral, once-daily Orladeyo in pediatric patients with HAE who are 2 to <12 years of age. The congress will take place in San Diego from 28 February – 3 March 2025.
BioCryst will present the following five abstracts on Sunday, 2 March from 9:45-10:45 am Pacific Time (PT) in Hall A at the San Diego Convention Center:
- HAE Attack Rates in Pediatric Patients 2 to <12 Years of Age with Prophylactic Berotralstat: Results from Interim Analysis of APeX-P; Poster #L55
- Real-World Attack Rates Before and After Berotralstat Initiation Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency (Type I/II) Stratified by Monthly Baseline HAE Attack Frequency; Poster #603
- Real-World Attack Rates Before and After Berotralstat Initiation Among Patients with Hereditary Angioedema without C1-Inhibitor Deficiency (HAE-nl-C1-INH) Stratified by Monthly Baseline HAE Attack Frequency; Poster #607
- Exploring the Role of Disease Burden, Treatment Effectiveness, and Administration Preference on Willingness of Patients With HAE to Change Long-Term Prophylaxis; Poster #608
- Patient-Reported Impact of Berotralstat as Long-Term Prophylaxis on Hereditary Angioedema Attack Frequency and Attack Severity; Poster #655
(Source: BioCryst)
12 February, 2025
BioCryst launches Orladeyo (berotralstat) in Portugal
BioCryst Pharmaceuticals announced that Infarmed in Portugal has recommended Orladeyo (berotralstat) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in eligible patients 12 years and older. With this recommendation, HAE patients in Portugal will have access to the first oral, once-daily therapy for the reduction of recurrent HAE attacks.
“The positive Infarmed recommendation of Orladeyo broadens access to modern prophylaxis, providing greater choice for prescribing physicians and potentially a better quality of life for HAE patients in Portugal,” said Charlie Gayer, chief commercial officer of BioCryst.
The Infarmed decision in Portugal follows the European Commission marketing authorization of Orladeyo in April 2021. To date, Orladeyo is licensed in 44 countries.
(Source: BioCryst)
13 February, 2025
European Commission approves CSL’s Andembry (garadacimab) for the prevention of recurrent attacks of hereditary angioedema (HAE)
CSL announced that the European Commission (EC) has approved Andembry (garadacimab), the first and only once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older. Andembry inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body.
Andembry reinforces CSL’s decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection.
Bill Mezzanotte MD, Executive Vice President and Head of R&D at CSL, said: “Andembry is a significant advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method. Andembry, CSL’s first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients. Thank you to all the colleagues, physicians and patients who contributed to this exciting milestone for HAE patients and CSL.”
Henrik Balle Boysen, President of HAE International (HAEi), said: “Garadacimab, a novel once monthly subcutaneous treatment that inhibits activated Factor XII, is a welcome addition to the HAE treatment landscape. People with HAE now have another choice for lessening the burden associated with this lifelong condition and realizing the community’s shared goal of experiencing life to the fullest.”
(Source: CSL)
20 February, 2025
CSL receives approval in Japan for Andembry (garadacimab) subcutaneous injection 200mg pens, a novel human anti-activated Factor XII monoclonal antibody for the prevention of acute attacks of hereditary angioedema (HAE)
CSL announced that it has received manufacturing and marketing approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for Andembry (garadacimab) subcutaneous injection 200mg Pens. The product is approved for the prevention of acute attacks of hereditary angioedema (HAE) and is the first pre-filled pen presentation for once-monthly subcutaneous administration for long-term prophylaxis of HAE. The approval in Japan follows additional recent approvals received in Australia, the United Kingdom, and the European Union.
Andembry is the first fully human monoclonal antibody in Japan designed to inhibit activated Factor XII (Factor XIIa), which initiates the cascade of events leading to angioedema at various sites of the body.
Bill Mezzanotte MD, Executive Vice President, Head of R&D at CSL, said: “Andembry represents a major advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term disease control through a patient-centric and convenient administration method. As CSL’s first approved recombinant monoclonal antibody discovered and developed entirely by CSL, Andembry underscores our more than 40-year commitment to HAE research and treatment optimization. This milestone is the result of decades of dedication, and we extend our gratitude to the colleagues, physicians and patients who made this possible for HAE patients and CSL.”
(Source: CSL)
20 February, 2025
Astria Therapeutics to present at upcoming American Academy of Allergy, Asthma and Immunology and World Allergy Organization Joint Congress
Astra Therapeutics announced that it will present three posters at the American Academy of Allergy, Asthma and Immunology (AAAAI) and World Allergy Organization (WAO) Joint Congress in San Diego, California on 2 March 2025.
- Markus Magerl MD, Professor of Dermatology and Allergy at the Charité Universitätsmedizin Berlin Institute of Allergology, will present the design of the global Phase 3 trial of navenibart in a presentation of poster number 617 titled, “ALPHA-ORBIT – A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Navenibart in Adolescent and Adult Participants with Type 1 and Type 2 Hereditary Angioedema (HAE).”
- Joshua S Jacobs MD, Medical Director of Allergy and Asthma Clinical Research Inc, will present attack severity reduction data from the ALPHA-STAR Phase 1b/2 trial of navenibart in a presentation of poster number 606 titled, “Treatment with Navenibart (STAR-0215) Reduces Attack Severity and Use of Rescue Medication in Patients with Hereditary Angioedema (HAE): Interim Results from the ALPHA-STAR Trial.”
- Chunxia Lily Zhao PhD, Director of Antibody Discovery at Astria Therapeutics, will present information on the profile of STAR-0310 in poster number 697 titled, “Preclinical Data Supporting the Differentiated Profile of STAR-0310, a Novel OX40 Antagonistic Monoclonal Antibody.”
All three presentations will take place in a poster session titled “Atopic Dermatitis, Contact Dermatitis, Urticaria, Angioedema” beginning at 9:45am Pacific Standard Time on 2 March 2025, in the Convention Center, Ground Level, Hall A.
(Source: Astria)
21 February, 2025
KalVista Pharmaceuticals to present new sebetralstat data at the American Academy of Allergy, Asthma & Immunology 2025 Annual Meeting
KalVista Pharmaceuticals announced that six abstracts have been accepted for presentation at the American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) 2025 Joint Congress taking place in San Diego, California from 28 February – 3 March 2025.
The following poster presentations will take place on Friday 28 February 28 2025 from 2:45–3:45pm Pacific Time (PT) in the Convention Center, Ground Level, Hall A:
- Barriers to Timely On-demand Treatment of Hereditary Angioedema Attacks in Italian Patients (#180): Mauro Cancian, Paola Triggianese, Pietro Accardo, Francesco Arcoleo, Donatella Bignardi, Caterina Colangelo, Francesco Giardino, Antonio Gidaro, Marica Giliberti, Maria Domenica Guarino, Paola Lucia Minciullo, Stefania Nicola, Francesca Perego, Riccardo Senter, Giuseppe Spadaro, Massimo Triggiani, Sherry Danese, Julie Ulloa, Vibha Desai, Paul Audhya, Andrea Zanichelli.
- Burden of Injectable On-Demand Treatment for Hereditary Angioedema Attacks in Adolescents (#185): Mauro Cancian, Paula Busse, Tariq El-Shanawany, Maeve O’Connor, Sinisa Savic, Paola Triggianese, Patrick Yong, Andrea Zanichelli, Sherry Danese, Julie Ulloa, Vibha Desai, Paul Audhya, Sandra Christiansen.
- Delays in the On-demand Treatment of Hereditary Angioedema Attacks and Associated Barriers Reported in Different Healthcare Systems (#187): Patrick Yong, Paula Busse, Timothy Craig, Tariq El-Shanawany, Padmalal Gurugama, Rashmi Jain, Maeve O’Connor, Cristine Radojicic, Sinisa Savic, James Wedner, Sherry Danese, Julie Ulloa, Vibha Desai, Paul Audhya, Sandra Christiansen.
The following late-breaking poster presentations will take place on Saturday 1 March 2025, from 9:45–10:45am PT in the Convention Center, Ground Level, Hall A:
- Study to Adjudicate Hereditary Angioedema with Normal C1INH Diagnoses in the PIONEER-HAE Database (#L14): Maeve O’Connor, Dana Withrow, Scott Milligan, Vibha Desai, Paul Audhya, Marc Riedl.
- Effectiveness of Sebetralstat for the On-demand Treatment of Laryngeal Hereditary Angioedema Attacks: Interim Analysis from KONFIDENT-S (#L15): Jonathan Bernstein, Emel Aygoren-Pursun, Vesna Grivcheva-Panovska, Inmaculada Martinez-Saguer, Danny Cohn, William Lumry, Marc Riedl, Andrea Zanichelli, James Hao, Michael Smith, Christopher Yea, Utpaul Audhya, Henriette Farkas.
The following oral presentation will take place on Saturday 1 March 2025, from 2:25–2:35pm PT in the Convention Center, Room 6C, Upper Level:
- On-demand Treatment Of Hereditary Angioedema Attacks With Sebetralstat In Adolescents: Pooled Analysis From KONFIDENT And KONFIDENT-S (#552): Danny Cohn, Aharon Kessell, Tamar Kinaciyan, Rand Arnaout, Jonathan Peter, H. James Wedner, Emel Aygoren-Pursun, Jonathan Bernstein, Henriette Farkas, William Lumry, Marcus Maurer, Marc Riedl, Andrea Zanichelli, James Hao, Matthew Iverson, Michael Smith, Christopher Yea, Utpaul Audhya, Fotios Psarros.
(Source: KalVista)
24 February, 2025
BioCryst reports upcoming key milestones
BioCryst Pharmaceuticals provided a corporate update.
Jon Stonehouse, President and Chief Executive Officer of BioCryst, said: “We ended 2024 with the strongest execution and performance in the company’s history, and this year is off to a fantastic start, with our pediatric label expansion for Orladeyo anticipated later this year.”
The company has received final reimbursement for Orladeyo in Portugal. Orladeyo is now reimbursed in all major countries in Western Europe, except the Netherlands, which is expected in 1H 2025.
Dr Helen Thackray, Chief R&D Officer of BioCryst, said: “There is a tremendous unmet need for an oral option for children with HAE, so we are excited to bring Orladeyo to children as young as two years old.”
The company is on track to submit a new drug application (NDA) in 2025 to the U.S. Food and Drug Administration (FDA) to expand the Orladeyo label to children with HAE aged 2 to 11 using an oral granule formulation. Additional regulatory filings are planned in global territories, including Europe, Japan and Canada. Orladeyo would be the first targeted oral prophylactic therapy for children with HAE.
(Source: BioCryst)
24 February, 2025
The European Medicines Agency (EMA) has approved an additional subcutaneous administration option for Takhzyro (lanadelumab) for patients aged 12 years and above with recurrent attacks of hereditary angioedema (HAE)
Takeda announced that the European Medicines Agency (EMA) has approved an additional 2 mL pre-filled pen option for Takhzyro (lanadelumab) for subcutaneous administration in adolescents (aged 12 years and above) and adult patients with hereditary angioedema (HAE).
The additional subcutaneous administration option expands Takeda’s offering in this space, showing dedication to the HAE community while providing individualized treatment options to support patients with a life-threatening disease, by helping to reduce HAE burden and improving their quality of life.
Irmgard Andresen, Global Medical Lead HAE at Takeda, said: “HAE affects an estimated 1 in 50,000 people worldwide and is often under recognised, under diagnosed and under treated. We welcome the swift approval by the EMA on this additional subcutaneous administration option. HAE patients 12 years and older now have an additional individualized treatment option available to them.”
(Source: Takeda)
24 February, 2025
BioCryst announces positive results from APeX-P trial for Orladeyo (berotralstat) in pediatric patients with hereditary angioedema aged 2 to <12 years
BioCryst Pharmaceuticals announced positive results from an interim analysis of the ongoing APeX-P clinical trial evaluating an oral granule formulation of once-daily Orladeyo (berotralstat) in pediatric patients with hereditary angioedema (HAE) aged 2 to <12 years.
Dr Helen Thackray, Chief Research and Development Officer of BioCryst, said: “We are pleased to share results from APeX-P, the largest trial to date evaluating a prophylactic therapy for HAE in patients 2 to <12 years old, which will be presented later this week in a late-breaking abstract at the AAAAI / WAO Joint Congress. Importantly, the data show the oral granule formulation of Orladeyo to be safe and well tolerated in the trial, with early and sustained reductions in monthly attack rates. We remain on track to submit our New Drug Application to the FDA this year, and we look forward to addressing a significant unmet need for children with HAE and their families.”
HAE Attack Rates in Pediatric Patients 2 to <12 Years of Age with Prophylactic Berotralstat: Results from Interim Analysis of APeX-P; Poster #L55
- Orladeyo was safe and well tolerated in APeX-P, with no new safety signals identified. Adverse events (AEs) were similar across all ages and weights.
- Orladeyo resulted in early and sustained reductions in monthly attack rates. The median (range) and mean (±SEM) monthly attack rates in the standard-of-care period were 0.96 (0–5.0) and 1.5 (±0.2) attacks/month, respectively. After one month of taking Orladeyo, median and mean monthly attack rates dropped to 0 (0-4.0) and 0.5 (±0.2), and the median monthly attack rate remained at 0 through month 12 (month 12 range: 0-1.7); the mean monthly attack rate at month 12 was 0.3 (±0.1).
- Eighty-three percent of participants experienced symptom onset before six years of age and 90 percent were diagnosed with HAE in the same timeframe.
(Source: BioCryst)
26 February, 2025
Swissmedic approves CSL Behring’s Andembry (garadacimab) for the prevention of recurrent attacks of hereditary angioedema (HAE)
CSL announced that the Swiss Agency for Therapeutic Products (Swissmedic) has granted marketing authorization to Andembry (garadacimab) for long-term prophylaxis of recurring attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. Andembry, a monoclonal antibody, inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body.
Emmanuelle Lecomte-Brisset, Global Head of Regulatory Affairs at CSL, said: “This approval from Swissmedic marks the fifth regulatory approval of Andembry for HAE, following recent approvals in Australia, the UK, the EU, and Japan. We are incredibly proud of these milestones, which reflect our decades-long commitment to delivering innovative medicines to the HAE community. Access and reimbursement negotiations are currently underway to ensure that Andembry is made available to eligible people with HAE in Switzerland.”
Dr Isabelle Dahinden, General Manager Switzerland, CSL Behring, said: “With the approval of Andembry, following Berinert IV (intravenous) and Berinert SC (subcutaneous), CSL Behring now offers an even more comprehensive portfolio for the management of HAE. This allows us to meet the diverse needs of people living with HAE, a community we have supported for more than 40 years.”
Dr Michael Haslauer, Country Medical Lead Switzerland, CSL Behring, said: “While there are current treatments available for HAE, individual needs and responses to treatment vary. Andembry offers people living with HAE a vital new treatment option that provides long-term control over their disease.”
(Source: CSL)
27 February, 2025
Astria Therapeutics initiates ALPHA-ORBIT Phase 3 pivotal trial of navenibart in hereditary angioedema
Astra Therapeutics announced the initiation of the ALPHA-ORBIT Phase-3 clinical trial of navenibart in people living with hereditary angioedema (HAE). Navenibart has the potential to provide rapid and sustained HAE attack prevention with a very low treatment burden and administration every 3 months (Q3M) and every 6 months (Q6M).
Christopher Morabito MD, Chief Medical Officer at Astria Therapeutics, said: “We believe that navenibart will deliver strong efficacy, low treatment burden, and favorable safety and tolerability and we are thrilled to have initiated our Phase 3 ALPHA-ORBIT trial to support that vision. The Phase 3 program is designed to enable options, providing patients and physicians with the potential to decide what works best for them by administering navenibart only 2 or 4 times per year.”
Dr Aleena Banerji, Clinical Director MGH Allergy and Immunology Unit, and a Principal Investigator for the ALPHA-ORBIT trial, said: “We understand from patients that it would be incredibly meaningful to have a therapy that would enable them to live their lives free from the limitations of HAE. Navenibart has demonstrated the potential to prevent HAE attacks with infrequent dosing, which could allow patients the freedom to spend less time managing their disease.”
ALPHA-ORBIT is a global, randomized, double-blind, placebo-controlled Phase 3 pivotal clinical trial to evaluate the efficacy and safety of navenibart over a 6-month treatment period in up to 135 adults and 10 adolescents (open label), with HAE Type 1 or Type 2. Adult patients will be randomized to receive one of three navenibart dose arms:
- An initial 600mg dose followed by 300mg Q3M
- 600mg Q6M
- 600mg Q3M, or placebo; adolescents will receive an initial 600mg dose followed by 300mg Q3M
The dose arms support the potential to provide patient-centered dosing flexibility to people with HAE. The primary endpoint is time-normalized monthly HAE attacks at 6 months, and a key secondary endpoint includes the proportion of participants who are attack-free at 6 months. Top-line results from the trial are anticipated in early 2027.
(Source: Astria)
3 March, 2025
Intellia Therapeutics highlights recent company progress
Intellia Therapeutics reported operational highlights.
John Leonard MD, Intellia President and Chief Executive Officer, said: “We are off to an excellent start in 2025 with renewed focus and strong operational execution. We are excited by the clinical data presented during the fourth quarter. Our Phase 1/2 results in HAE suggest that NTLA-2002 could represent a functional cure for patients with HAE – for the first time a patient has the potential to be both free from attacks and free from chronic therapy.”
Recent operational highlights in hereditary angioedema (HAE):
- In January, Intellia announced the first patient was dosed with NTLA-2002 in the global Phase 3 HAELO study. The Company expects to complete enrollment in the second half of 2025.
- The Company plans to submit a Biologics License Application in the second half of 2026 to support plans for a U.S. launch in 2027.
- Intellia expects to present longer-term data from the ongoing Phase 1/2 study in 2025. The data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.
(Source: Intellia)
3 March, 2025
KalVista Pharmaceuticals shares latest sebetralstat findings at the American Academy of Allergy, Asthma & Immunology 2025 Annual Meeting
KalVista Pharmaceuticals announced the presentation of novel sebetralstat data related to laryngeal hereditary angioedema (HAE) attacks and adolescents with HAE at the American Academy of Allergy, Asthma & Immunology (AAAAI) / World Allergy Organization (WAO) 2025 Joint Congress taking place in San Diego, California from 28 February – 3 March 2025.
Ben Palleiko, CEO of KalVista, said: “The growing body of data from the KONFIDENT-S study consistently demonstrate that sebetralstat enabled early treatment and fast symptom relief from HAE attacks, regardless of age, attack location, or severity. This is especially critical for vulnerable populations, such as those experiencing laryngeal attacks or adolescents whose only approved options are injectable on-demand treatments. Sebetralstat, if approved, would be the first oral on-demand treatment for HAE attacks, with the potential to address some of the most significant unmet needs in HAE.”
Effectiveness of Sebetralstat for the On-demand Treatment of Laryngeal Hereditary Angioedema Attacks: Interim Analysis from KONFIDENT-S
- 32 laryngeal attacks were treated with sebetralstat (14 September 2024 cutoff)
- Median time to treatment with sebetralstat: 11.5 minutes after attack onset
- Median time to beginning of symptom relief: 1.27 hours
- 96% of those achieving beginning of symptom relief within 12 hours did so with a single dose
- No reports of difficulty swallowing film-coated tablet
On-demand Treatment of Hereditary Angioedema Attacks with Sebetralstat In Adolescents: Pooled Analysis From KONFIDENT And KONFIDENT-S
- 149 attacks were treated with sebetralstat across KONFIDENT/KONFIDENT-S (14 September 2024 cutoff)
- Median time from attack onset to treatment: 4 minutes
- Safety and efficacy consistent with adults; no serious adverse events or adverse events leading to discontinuation
(Source: KalVista)
3 March, 2025
Pharvaris presents long-term clinical data of deucrictibant for the prevention and treatment of HAE attacks at the 2025 AAAAI/WAO Joint Congress
Pharvaris highlighted safety and efficacy data of deucrictibant, which is currently being evaluated in two pivotal Phase 3 studies, following long-term dosing in the prophylactic and on-demand settings at the American Academy of Allergy, Asthma, & Immunology’s Annual Scientific Meeting (AAAAI) and World Allergy Organization (WAO) Joint Congress, which was held from 28 February – 3 March 2025, in San Diego, California.
Peng Lu MD, PhD, Chief Medical Officer of Pharvaris, said: “Topline results of deucrictibant in both prophylactic and on-demand randomized clinical trials substantiate our belief in the mechanism and molecule to provide choice to those living with HAE; continued analyses of clinical outcomes and health-related quality of life measures from the extension studies, such as these presented at the 2025 AAAAI/WAO Joint Congress, help solidify our confidence in deucrictibant’s ability to meet existing unmet needs in the HAE community. The safety and efficacy data of deucrictibant following long-term dosing in a prophylactic clinical setting is especially noteworthy. Participants experienced a median of zero days with attack symptoms each month, and enhanced quality-of-life, specifically within the observed HRQoL domains of the greatest improvement— ‘functioning’ and ‘fear and shame’ — which are particularly relevant to people living with HAE.”
Dr Lu continued: “Additionally, we understand from the HAE community that there is a desire for an oral, on-demand therapy that can rapidly and completely treat any type of attack with a single dose. Although the sample size is small, in line with the rarity of these types of attacks, we are pleased to share data from seven upper airway, including laryngeal, attacks that were treated with deucrictibant; these safety and efficacy findings were consistent with those seen in the 328 non-upper airway attacks treated with deucrictibant showing rapid and complete symptom resolution with a single dose. The encouraging data from these extension studies further underscore our opportunity to potentially introduce a therapeutically meaningful oral therapy for the prevention and treatment of HAE attacks, the profile of which we aim to confirm with Phase 3 data.”
Berndt Modig, Chief Executive Officer of Pharvaris, said: “This year is paramount to Pharvaris as we continue clinical development of deucrictibant to help address unmet needs for those living with bradykinin-mediated angioedema. Pharvaris is committed to generating robust clinical data to build a compelling package supporting deucrictibant’s efficacy and safety profile. Our team is focused on the execution of two Phase 3 clinical studies in HAE, the expansion of our pipeline into AAE, and preparations for commercialization of deucrictibant pending regulatory submission and approval; we have significant resources in place to support these strategic investments and provide value for our shareholders.”
(Source: Pharvaris)
25 March, 2025
KalVista Pharmaceuticals Announces Early Completion of Enrollment in KONFIDENT-KID Pediatric HAE Trial
KalVista Pharmaceuticals announced the completion of enrollment in the open-label KONFIDENT-KID clinical trial of sebetralstat in pediatric patients between the ages of two and 11 with hereditary angioedema (HAE).
Ben Palleiko, CEO of KalVista, said: “We’re proud to share that we achieved target enrollment in our KONFIDENT-KID trial a full year ahead of schedule and expanded the trial size due to overwhelming interest. The high level of participation from families living with HAE underscores the significant need for an oral treatment option for this population. Even with this larger trial size, we anticipate that initial results will be shared later this year, with an sNDA expected to be filed by mid-2026.”
Originally designed to enroll 24 pediatric patients, the trial was met with high demand and will ultimately include approximately 36 patients between the ages of two and 11 across seven countries in North America, Europe, and Asia. KONFIDENT-KID will collect safety, pharmacokinetic, and efficacy data for up to one year and features a proprietary pediatric oral disintegrating tablet (ODT) formulation of sebetralstat.
(Source: KalVista)
HAEi Around the World
Currently, there are HAE Member Organizations in 103 countries. You will find a great deal of vital information on the HAE representations around the globe at haei.org, and the world map will provide you with contact information for the Member Organizations, ACARE centers, hospitals, physicians, and available medication.
The information on haei.org is updated as soon as HAEi receives fresh data from the national Member Organizations.
Global Perspectives · Issue 1/2025 · April 2025
Magazine Staff: Henrik Balle Boysen, President; Deborah Corcoran, Director Research; Stuart Mayell, Editor in Chief, Global Perspectives; Rikke Sørensen, Manager, Communication, Design, and Graphics
Subscription: If you would like to subscribe to our magazine, please send an email to info@haei.org or register directly at haei.org
PDF version of the magazine is available on our website – click here to see the PDF and earlier editions of Global Perspectives.
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Who we are
HAE International is a global non-profit network of member organizations dedicated to raising awareness of hereditary angioedema and improving the lives of people with HAE.
Contact us
HAE International (HAEi)
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info@haei.org
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