Data on long-term HAE disease control with donidalorsen unveiled at 2025 ACAAI Annual Scientific Meeting
The latest data on a new medicine for HAE, donidalorsen (brand name Dawnzera), was revealed at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting, Orlando, USA, 6-10 November 2025.
The following is a summary of some key findings:
- Donidalorsen showed durable efficacy, demonstrating 94% overall mean HAE attack rate reduction at one year in an open-label extension study
- Patients in a study looking at treatment switching, achieved a 68% improvement in mean HAE attack rate compared to prior prophylactic therapy one year after switching to donidalorsen
- Long-term efficacy and safety maintained up to four years in the Phase 2 open-label extension study
Donidalorsen was recently approved by the US Food and Drug Administration for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older.
Kenneth Newman MD, Senior Vice President, Clinical Development at Ionis, said: “These new long-term data support our belief that donidalorsen, which is now available to people living with HAE in the US, is well-positioned to transform the treatment paradigm for HAE. The latest results from across our clinical studies support the strong and durable efficacy and safety profile of donidalorsen. Presentations at the congress offer patients and physicians valuable insights to support informed treatment decisions, including when transitioning to donidalorsen from a previous prophylactic therapy.”
(Source: Ionis)