A global movement in action

After the short movement break, the first of a packed day of dedicated patient leader and advocacy sessions commenced in the main auditorium.

On stage were Fiona Wardman, Executive Vice President (EVP) and Chief Advocacy Officer, and Jørn Schultz-Boysen, EVP and Chief Operating Officer. Both took the opportunity to highlight some of the hotly anticipated sessions on the agenda, including spotlight sessions on educating and engaging young people in advocacy through the HAEi LEAP program and an afternoon of breakout sessions where advocates from similar geographies will be able to pool their knowledge and share experiences to improve the lives of those they serve.

Before all that, Jørn and Fiona welcomed their Executive colleagues, Henrik and Tony back to the stage.

‘We honor the persistent commitment to advocacy that everyone in this room brings to the table.’

– Tony Castaldo, HAEi CEO and Chairman of the Board

Telling the assembled delegates how incredible they are, Tony made clear that the entire community knows they are the ones who show up when it counts, fighting for access to HAE therapies and making sure no patient feels alone or overlooked.

The family of Member Organizations continues to grow, with the network now spanning 106 countries worldwide. HAEi, Tony remarked, recently welcomed new advocacy organizations in Vietnam, Trinidad and Tobago, Niger, Namibia, and Sri Lanka. Still, every member country, whether newly established or not, represents a story of real courage and persistence.

That network of Member Organizations isn’t just growing, but succeeding. According to Henrik, there are compelling stories of advocacy in action from all corners of the world. He highlighted just a few to the audience:

  • In Tunisia, an HAEi workshop brought together 70 patients and caregivers for the first time
  • In Pakistan, an HAEi-supported medical webinar reached more than 40 physicians who had never received HAE training
  • In Panama, a joint effort between the Member Organization, physicians, and HAEi led to the country approving its first official HAE treatment guideline, and each hospital in the country is now putting the guideline into practice
  • In Poland, sustained advocacy secured reimbursement for a new preventive therapy
  • In Argentina, a coordinated community effort reversed a negative government recommendation on access to a critical medicine
  • In Armenia, patients who had never had access to modern medication enrolled in a clinical trial that opened treatment options for the very first time

All these advances were, according to Henrik, the result of people like those in the room, who exhibited a never-give-up attitude and spirit.

Tony and Henrik shared an overview of the current treatment landscape, which they reminded the audience makes HAE a leader in rare diseases and something everyone involved in advocacy should be extremely proud of. They outlined the following medicines:

  • CSL Behring offers Berinert and Haegarda, both plasma-derived C1-inhibitor therapies for on-demand treatment and prevention of attacks, respectively
  • Takeda offers Cinryze, another C1 inhibitor concentrate for on-demand treatment and prevention, as well as Firazyr for on-demand treatment. Additionally, the company offers Takhzyro for routine prevention and Kalbitor, which is an acute treatment primarily available in the United States
  • From Pharming, there is Ruconest, a recombinant C1 inhibitor concentrate to treat attacks, which is also primarily available in the United States
  • BioCryst produces Orladeyo, which is an oral, once daily, preventive therapy
  • Icatibant, which is a generic form of Firazyr, is available from a variety of manufacturers

Additionally, the community saw a remarkable three new medicines become available in 2025. These were:

  • CSL Behring’s Andembry, which inhibits Factor 12 and is given once a month for the prevention of HAE attacks
  • Ionis launched Dawnzera, which works to prevent HAE attacks by targeting pre-kallikrein with dosing every four or eight weeks
  • KalVista made Ekterly available, which is an oral plasma kallikrein inhibitor for the on-demand treatment of HAE attacks

Adding to all this good news, five more companies are developing new HAE therapies:

  • ADARx is progressing a medicine called onvuzosiran, which uses short-interfering RNA (siRNA), in trials to prevent attacks with dosing every three or six months
  • Argo Biopharma’s investigational new treatment, known by the codename BW20805, is another siRNA therapy targeting pre-kallikrein to prevent attacks, with dosing every three and six months being tested
  • Astria, now part of BioCryst, is developing navenibart, a plasma kallikrein inhibitor currently tested for attack prevention, taken every three or six months
  • Intellia is currently testing lonvoguran ziclumeran, a gene-editing therapy based on CRISPR-Cas9 technology. This therapy, which is in Phase-3 trials at present, aims to inactivate the gene that makes pre-kallikrein with the goal of long-term attack prevention
  • Pharvaris is developing deucrictibant, an oral therapy trialed for both on-demand treatment and attack prevention.

These companies, Tony made clear, are here at this conference for a reason: “They are here because they want to hear from you.” Tony encouraged everyone present to engage with the company representatives, share the unique needs of people with HAE, and ask for commitment and support for their individual organizations.

Tony and Henrik concluded by warmly welcoming everyone present and said how much they looked forward to personally greeting everyone over the coming two days.