In addition to a wide range of case reports, reviews, and small series, here are summaries of recently published HAE-related scientific papers. Data search undertaken 5 January 2026. The source used is the National Library of Medicine (NLM).
Sebetralstat: First approval
Blair HA
Writing in the journal Drugs, the author reviews the chemical and clinical aspects of sebetralstat and its regulatory pathway, as it was recently approved for use in the United States.
(Drugs, November 2025)
Editorial: Bradykinin and histamine mediated angioedema
Patil AD, et al
The authors introduce a collection of 11 research and review articles, which look at both histamine-mediated and bradykinin-mediated angioedema.
(Frontiers in Allergy, November 2025)
Long-term safety and effectiveness of sebetralstat: Interim analysis of KONFIDENT-S open-label extension
Farkas H, et al
This clinical trial looked at the long-term use of sebetralstat in people with HAE 12 years old and over. The authors found that side effects occurred in 9.5% of participants, but none were serious. The authors conclude that the oral formulation of sebetralstat enabled compliance with treatment guidelines and that safety and effectiveness were consistent with earlier trial results.
(Journal of Clinical Immunology: In Practice, November 2025)
Therapeutic advances in hereditary angioedema: A focus on present and future options
Uminski K, et al
The authors review the progression of therapeutic options for the management of HAE, and aim to summarize current advances and what the future could look like in terms of personalized and patient-centred care.
(Advances in Therapy, 11 October 2025)
Status quo and future developments in the diagnosis and treatment of hereditary angioedema
Recke A
The author aims to provide an overview of the clinical presentation, diagnosis, and treatment of HAE.
(Journal der Deutschen Dermatologische Gesselschaft, December 2025)
Garadacimab in hereditary angioedema due to normal C1INH with F12/PLG mutations
Cohn DM, et al
The authors conducted a clinical trial to determine whether long-term prophylaxis with garadacimab would help patients with HAE with normal C1 caused by mutations in Factor 12 (FXII) or plasminogen (PLG). They conclude that the safety profile is favorable and that there was evidence of efficacy in two of the three patients with HAE-FXII. A reduction in attack rates was observed in one of the three patients with HAE-PLG.
(Journal of Allergy and Clinical Immunology, December 2025)
Lanadelumab’s impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence
Zanichelli A, et al
This study aimed to assess the effectiveness of lanadelumab in patients with HAE by examining the frequency of attacks prior to treatment. The authors used real-world data from 152 patients with low, moderate, high, and very high disease activity. The authors concluded that, regardless of baseline disease activity, attack rates among patients treated with lanadelumab were low, and all patient groups experienced improvements in quality of life and disease control.
(Annals of Allergy, Asthma and Immunology, November 2025)
Leveraging lanadelumab: A phase 4 evaluation of hereditary angioedema by attack rate subgroup
Tarbox JA
This editorial accompanies the above paper by Zanichelli. In it, the author examines data from the new study and discusses the challenges related to the cost of targeted therapies. The author calls for greater partnership between physicians, patients, governments, and the pharmaceutical industry to bridge access gaps.
(Annals of Allergy, Asthma and Immunology, November 2025)
Insights from the first 820 patients from the Brazilian multicenter registry of hereditary angioedema: The key role of genetic testing and targeted therapies
Ferriani MPL, et al
The authors aimed to identify knowledge and management gaps relating to HAE in Brazil. To do this, a multicenter HAE registry was established. 820 patients were enrolled. The authors were able to conclude that Brazilian patients with HAE share common aspects with global patients, including predominance in women, and that HAE due to C1 inhibitor deficiency is the most common subtype. However, genetic testing also identified that 19.4% of patients in the registry had HAE due to Factor 12 mutation. The authors also indicate that access to first-line therapies for long-term prevention of HAE attacks remains limited.
(Journal of Clinical Immunology: In Practice, November 2025)
Delays and barriers related to the treatment of hereditary angioedema attacks in Italy
Cancian M, et al
In a letter to the editor, the authors provide details of their research into barriers to early on-demand (OD) treatment of HAE attacks. The authors found that the most common patient-reported reasons for delaying OD therapy are not unique to Italian patients, and that one in four survey respondents cited administration-related barriers leading to treatment delays.
The authors note that to improve patient outcomes, barriers to OD therapy should be proactively addressed. One way would be to better educate patients on the importance of recognizing early attack symptoms and shifting perception of ‘early treatment’.
(Allergy, November 2025)
French protocol for the diagnosis and management of hereditary angioedema
Boccon-Gibod I, et al
The authors present a French protocol for the diagnosis and management of bradykinin-mediated HAE.
(La Revue de Médecine Interne, December 2025)
C1 inhibitor: From complement system to bradykinin angioedema
Defendi F, et al
The authors reviewed current research into diagnostic and prognostic markers for bradykinin-mediated angioedema. They also address the link between C1 inhibitor deficiency and chronic comorbidities.
(Current Opinion in Immunology, December 2025)
CRISPR-Cas9 gene editing for hereditary angioedema: Current treatments and emerging therapies
Jalal L, et al
In this review, the authors summarize what is currently known about HAE treatment and the existing clinical evidence supporting the safety and efficacy of NTLA-2002 (also known as lonvoguran ziclumeran or lonvo-z), a potential therapy for HAE.
(Annals of Medicine and Surgery (London), November 2025)
Oral berotralstat for hereditary angioedema prophylaxis in patients aged 2 to <12 years: APeX-P interim results
Bernatoniene J, et al
This clinical study evaluated oral berotralstat for the long-term prevention of HAE in pediatric patients. The authors concluded that oral berotralstat was well tolerated and resulted in early and sustained reductions in HAE attack rates.
(Annals of Allergy, Asthma and Immunology, December 2025)
Berotralstat, the first oral prophylaxis for hereditary angioedema in children aged 2 to 12 years: The kids are alright!
Levy DS
This editorial, linked to the above research by Bernatoniene and colleagues, reviews the current treatment landscape for HAE in children. The author suggests that on-demand and prophylactic options are limited compared to those available to adults. The burden of on-demand therapy is suggested to be significant, with half of adolescents reporting being anxious about the use of on-demand therapy for their HAE attacks. There is also a mean time to treatment of seven hours, with a delay to treatment resulting in prolonged duration of HAE attacks.
(Annals of Allergy, Asthma and Immunology, December 2025)
Treatment of hereditary angioedema with plasma-derived C1 inhibitor: A review
Martinez-Saguer I, et al
In this review, the authors provide an in-depth overview of the efficacy and safety of plasma-derived C1 inhibitor replacement therapy as a first-line treatment for on-demand and short and long-term prophylaxis in patients with various types of HAE.
(Clinical and Translational Allergy, December 2025)
Long-term prophylactic treatment with deucrictibant for angioedema due to acquired C1-inhibitor deficiency
De Lange M, et al
The authors present the findings of a small clinical trial evaluating the long-term safety and efficacy of deucrictibant as prophylaxis in patients with acquired C1 inhibitor deficiency (AAE-C1INH). They conclude that deucrictibant extended-release tablets effectively prevented angioedema attacks in patients with AAE-C1INH, with no safety concerns.
(Journal of Allergy and Clinical Immunology, December 2025)
Cardiac and vascular autonomic control in patients with hereditary angioedema
De Maria B, et al
In HAE, due to C1 inhibitor deficiency, the body’s ability to regulate vascular permeability is key. The authors compared the cardiovascular responses of people with HAE and healthy matched controls across different body positions (e.g., lying down or lifted to a near-standing position). The authors measured heart rate and blood pressure throughout the tests. They conclude that older HAE-C1INH patients display altered vascular regulation, with an exaggerated response when upright. There appeared to be no difference in outcomes between patients on long-term prophylaxis or not.
(Frontiers in Physiology, December 2025)
Exposure-response analysis of donidalorsen for the treatment of hereditary angioedema
Singh P, et al
Using a series of simulations, the authors aimed to predict the efficacy of potential dosing regimens of donidalorsen to prevent HAE attacks. The authors conclude that their simulations support the efficacy of dosing every month, and every two months. They believe that switching to dosing every two months could be a viable approach for patients who are attack-free on a monthly dosing regimen.
(Clinical and Translational Science, November 2025)
Plasma kallikrein inhibitors for multiple disorders: Current advances and perspectives
Liu H, et al
The authors review the biological functions of plasma kallikrein and the most recent clinical advances in agents targeting this protease, including in HAE.
(Journal of Medicinal Chemistry, October 2025)
Perinatal management and clinical outcomes of hereditary angioedema in Japan: A case series and comprehensive literature review
Iino K, et al
The authors look at a small number of cases of women with type 1 HAE giving birth, in order to better understand how to manage HAE in perinatal women. The authors conclude that their findings indicate that perinatal care should be personalized for individual patients, and that short-term prophylaxis with plasma-derived C1 inhibitor should be the cornerstone of enhancing maternal safety and providing psychological reassurance.
(Journal of Dermatology, December 2025)
On-demand treatment of hereditary angioedema attacks: Patient-reported utilization, barriers, and outcome
Yi-Hsuan Tu, et al
The authors respond to a recent paper by Christiansen and colleagues that examines treatment behaviors among people with HAE. The authors offer suggestions for building on their work.
(Annals of Allergy, Asthma and Immunology, December 2025)
Common features of rare disease patients in the emergency department: A systematised literature review
Pflock S, et al
The authors investigated the common symptoms shared by a range of rare diseases, including HAE, to better understand how emergency department doctors can address undiagnosed rare diseases. The authors conclude that there is limited knowledge of rare diseases in the emergency department. There are common features among rare disease patients that could be used to identify those at increased risk and facilitate early detection and treatment.
(Orphanet Journal of Rare Diseases, November 2025)
Sebetralstat: A paradigm shift in hereditary angioedema management
Qadri M, et al.
The authors, writing in a letter to the editors, draw attention to the recent approval of sebetralstat by the US FDA. They indicate their belief that this could fundamentally alter the way patients with HAE are cared for. They believe that sebetralstat offers a desirable alternative for patients who presently rely on injectable medications, especially where these might not be easily accessible during an attack.
(Annals of Medicine and Surgery (London), October 2025)
A Phase 1 randomized study: Garadacimab pharmacokinetics, safety, and tolerability after administration via autoinjector/pre-filled pen versus pre-filled syringe in healthy participants
Glassman F, et al
The authors conducted a trial, using healthy volunteers, that compared the safety and how quickly the body absorbed garadacimab when a new auto-injector / pre-filled pen (AI/PFP), or an existing pre-filled syringe was used. The authors conclude that the AI/PFP had a consistent safety and tolerability profile, similar to that of the existing pre-filled syringe, providing at-home convenience for patients and physicians.
(The Journal of Clinical Pharmacology, January 2026)