In a significant development for patients suffering from hereditary angioedema (HAE), the U.S. Food and Drug Administration (FDA) approved three new treatments within a span of just three months in 2025. The approved therapies include garadacimab-gxii (Andembry) in June, sebetralstat (Ekterly) in July, and donidalorsen (Dawnzera) in August. HAE, a rare genetic condition that can lead to unpredictable and potentially life-threatening swelling attacks, has garnered increased attention from both medical professionals and patient advocacy groups.
The approvals mark a pivotal moment in the treatment landscape for HAE, which affects approximately 1 in 50,000 individuals globally, translating to around 6,000 patients in the United States. The challenges associated with diagnosing HAE often lead to delays in treatment, with some reports indicating that up to 90% of HAE-related deaths occur in patients who remain undiagnosed.
Daniel Soteres, MD, PhD, from the University of Colorado Health Sciences Center, emphasized the impact of these new options, stating, “Every patient has an individual set of values, an individual health literacy. Their needs change over time.” He highlighted the importance of having multiple treatment options available for patients of different ages and health profiles.
New Treatment Options Address Critical Needs
The recent approvals bring the total number of FDA-approved treatments for HAE to twelve, enhancing the ability of healthcare providers to tailor therapies to individual patient needs. Prior to these approvals, there were nine available options, including four preventive therapies and four for acute attacks.
The newly approved garadacimab-gxii is particularly noteworthy as it targets factor XIIa for prophylactic use, marking a first in HAE treatment. Approved on June 16, 2025, it is designed for adults and children aged twelve years and older. This treatment offers a once-monthly dosing regimen through a citrate-free, subcutaneous autoinjector, thus improving patient convenience.
In clinical trials, garadacimab-gxii demonstrated impressive results: 62% of treated patients remained attack-free, and there were over 99% reductions in overall HAE attacks compared to placebo. The pivotal data was gathered from the phase 3 VANGUARD trial, which solidified the drug’s efficacy.
On July 7, 2025, the FDA approved sebetralstat, marking the first oral, on-demand therapy for acute HAE attacks. The approval was based on phase 3 KONFIDENT trials, which indicated that patients experienced relief starting at a median time of 1.79 hours after treatment initiation. The ability to administer this oral therapy may greatly enhance treatment accessibility for both adult and pediatric patients.
Another revolutionary treatment, donidalorsen, received FDA approval on August 21, 2025. This RNA-targeted medication has shown to significantly reduce HAE attack rates, with studies indicating an 81% reduction in monthly attacks among patients. The data from the OASIS-HAE trial demonstrated substantial efficacy, further validating the need for such innovative therapies.
Addressing Patient Burdens and Future Needs
Experts attending the 2025 American College of Allergy, Asthma, & Immunology (ACAAI) Annual Scientific Meeting confirmed that children with HAE often face inconsistent emergency care, which can negatively affect their long-term outlook. A growing body of research indicates that the impact of HAE begins early in childhood, with many patients developing symptoms before the age of 20.
Raffi Tachdjian, MD, MPH, from UCLA Health, remarked on the rapid advancements in the field, stating, “In the span of just three months, there were three new treatment modalities approved in HAE, which is mind-blowing.” He reflected on the progress made since the introduction of modern treatments for HAE, noting that the developments offer hope for better disease management.
Despite these advancements, there remains a critical need for treatments specifically designed for children under twelve, as current options are limited. As highlighted by experts like Michael Manning, MD, a specialist in allergy and immunology, addressing the needs of younger patients is paramount. Manning noted, “HAE is really a disease of childhood,” emphasizing that a large percentage of patients show symptoms early in life.
The recent approvals signal a transformative period in the management of hereditary angioedema, offering new hope for patients who have long faced a complex and often debilitating condition. As research continues, experts are optimistic that further innovations will emerge, potentially expanding treatment options and enhancing patient outcomes.
