The landscape of treatment for hereditary angioedema (HAE) has transformed rapidly in 2025, marked by the US Food and Drug Administration’s (FDA) approval of three new therapies within a short span of just three months. These groundbreaking treatments—garadacimab-gxii (Andembry), sebetralstat (Ekterly), and donidalorsen (Dawnzera)—represent a significant advancement in managing this rare genetic condition that causes recurrent swelling attacks.
On June 16, 2025, the FDA approved garadacimab-gxii, notable for being the first treatment aimed at factor XIIa in adults and children aged 12 and older. This therapy offers a unique once-monthly dosing regimen and is administered via a subcutaneous autoinjector. According to pivotal data from the phase 3 VANGUARD trial, garadacimab-gxii allowed 62% of treated patients to remain attack-free, achieving a remarkable reduction of over 99% in the frequency of HAE attacks compared to placebo.
The momentum continued with the approval of sebetralstat on July 7, 2025. This treatment is distinguished as the first oral, on-demand option for acute HAE attacks, applicable for patients aged 12 and older. Reported trial results indicate that treatment can commence as quickly as four minutes after an attack begins, with significant symptom relief typically occurring within less than two hours.
Finally, donidalorsen received FDA approval on August 21, 2025, targeting prekallikrein to prevent HAE attacks in similar age groups. Data from the phase 3 OASIS-HAE trials revealed that donidalorsen reduced monthly attack rates by 81% for those receiving treatment every four weeks. A substantial 91% of patients on this regimen reported achieving good disease control.
Impact of New Treatments on HAE Management
Hereditary angioedema affects approximately 1 in 50,000 individuals globally, with around 6,000 cases reported in the United States. The condition is often associated with delayed diagnosis, complicating timely treatment. A report from May 2025 highlighted that up to 90% of HAE-related deaths occur in undiagnosed patients who experience asphyxiation during attacks.
At the 2025 American College of Allergy, Asthma, & Immunology Annual Scientific Meeting in Orlando, Florida, experts, including Daniel Soteres, MD, PhD, emphasized the significant burden HAE places on patients and their families. Soteres remarked, “Every patient has an individual set of values… Having multiple options available for these patients is important.” This sentiment highlights the need for a diverse array of therapeutic options tailored to the varying needs of patients at different life stages.
With the introduction of these new therapies, the total number of FDA-approved options for managing HAE has risen to twelve. Previously, there were only nine treatments available, which included both preventive and acute therapies. These therapies primarily function by either replacing C1 inhibitor proteins or blocking bradykinin, with administration routes spanning from intravenous to oral.
The recent approvals reflect a concerted effort over the past two decades to enhance the treatment landscape for HAE. Raffi Tachdjian, MD, MPH, from UCLA Health, expressed pride in the rapid progress, stating, “In the span of just three months, there were three new treatment modalities approved in HAE… it’s really a testament to all the work that’s gone into [the field].”
The Future of HAE Treatments
While the new approvals have sparked optimism among healthcare professionals, there remains a pressing need for treatments specifically designed for children under the age of 12. Experts highlighted that symptoms of HAE frequently appear much earlier than previously recognized, often as young as five years old, with some reports indicating onset as early as two.
Dr. Michael Manning, an allergy and immunology specialist from Arizona, noted, “HAE is really a disease of childhood. Eighty-five percent of patients develop symptoms before the age of 20.” This underscores the urgency for more pediatric-specific therapies to manage the disease effectively in younger patients.
The focus is also on addressing barriers that affect treatment accessibility, including the portability of medications and concerns for patients in rural areas or those who prefer less frequent dosing. As the field continues to evolve, the introduction of treatments that provide greater flexibility and improved patient experience will be crucial.
The recent FDA approvals signify a pivotal moment for those affected by HAE, offering hope for enhanced management options and improved quality of life. As Dr. Manning aptly stated, “The more we know, and the better we get at treating this, the better we get at finding a medication that is going to reach that goal of total control.”
