A New Era in HAE Management
For decades, the treatment of hereditary angioedema (HAE) was limited, and many patients experienced significant burden from painful, unpredictable, and sometimes life-threatening swelling attacks. In 2025, the therapeutic landscape for HAE expanded significantly with the approval of several new, innovative drugs. These new options offer patients more choices in how they manage their condition, from the convenience of an oral pill to less frequent, targeted injectable therapies.
Andembry (garadacimab): A Targeted Prophylactic
Andembry (garadacimab) is a prophylactic treatment approved for patients aged 12 and older. It targets activated factor XIIa (FXIIa), a key protein in the HAE swelling cascade. By inhibiting FXIIa, Andembry aims to prevent swelling episodes at their origin. It is administered as a once-monthly subcutaneous self-injection. Clinical trials showed that a significant percentage of patients treated with Andembry remained attack-free, with a substantial reduction in the median attack rate compared to placebo. {Link: patientworthy.com https://patientworthy.com/2025/07/17/fda-approves-ekterly-a-new-oral-option-for-hereditary-angioedema-attacks/}
Ekterly (sebetralstat): The First Oral On-Demand Therapy
Ekterly (sebetralstat) is the first and only oral on-demand treatment for acute HAE attacks in adults and pediatric patients aged 12 and older. Ekterly works by inhibiting plasma kallikrein. Its approval offers patients a convenient non-injectable option for rapid symptom relief, enabling early intervention wherever they are. This is particularly beneficial for those with needle phobia.
Dawnzera (donidalorsen): An RNA-Targeted Prophylactic
Dawnzera (donidalorsen) is an RNA-targeted medicine approved for HAE attack prevention in patients 12 and older. It targets plasma prekallikrein (PKK), a protein involved in the inflammatory process of HAE attacks. Dawnzera is administered via subcutaneous injection either once every four or eight weeks. Studies demonstrated that the four-week dosing schedule significantly reduced monthly attack rates compared to placebo, with sustained reductions observed in an open-label extension study.
Comparison of New HAE Treatments
| Feature | Andembry (garadacimab) | Ekterly (sebetralstat) | Dawnzera (donidalorsen) |
|---|---|---|---|
| Drug Type | Prophylactic (Preventive) | On-Demand (Acute Attack) | Prophylactic (Preventive) |
| Administration | Once-monthly subcutaneous injection | Oral capsule | Subcutaneous injection (every 4 or 8 weeks) |
| Mechanism | Targets activated factor XIIa (FXIIa) | Plasma kallikrein inhibitor | RNA-targeted inhibitor of plasma prekallikrein (PKK) |
| Indication | Prevent attacks in patients 12+ | Treat acute attacks in patients 12+ | Prevent attacks in patients 12+ |
| Primary Benefit | Infrequent dosing, high efficacy | Oral convenience, rapid intervention | Less frequent dosing than older prophylactics |
Implications for Patient Care
The introduction of these new HAE treatments provides greater flexibility in managing the condition. Patients and healthcare providers can now select therapies based on individual needs and preferences. Prophylactic options like Andembry and Dawnzera offer infrequent dosing for those with frequent attacks, while Ekterly provides the convenience of an oral option for treating acute attacks early. This expanded range of targeted and more convenient treatments represents a significant step forward in improving the lives of individuals with HAE.
Conclusion
The 2025 FDA approvals of Andembry, Ekterly, and Dawnzera have significantly broadened the treatment options for hereditary angioedema. These new drugs offer distinct mechanisms of action and administration methods, providing more choice and convenience for patients. The availability of different options empowers patients to manage their HAE more effectively. For more information, visit the {Link: US HAE Association https://www.haea.org/}
