Ionis Pharmaceuticals and Otsuka Pharmaceutical announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has granted a positive opinion for DAWNZERA (donidalorsen). The therapy is intended for the routine prevention of recurrent hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older. This recommendation now moves to the European Commission for a final approval decision, marking an important step toward making the therapy available across the European Union.
The CHMP’s assessment is supported by results from the Phase 3 OASIS-HAE and OASISplus clinical studies. In these trials, DAWNZERA showed consistent and meaningful reductions in mean monthly HAE attack rates. The therapy demonstrated sustained benefits across multiple measures of disease burden, including performance when self-administered through an autoinjector. These findings highlight its potential to offer reliable, long-term protection against unpredictable swelling episodes that significantly impact patient quality of life.
Hereditary angioedema is a rare genetic disorder characterized by episodic and sometimes severe swelling affecting various parts of the body, such as the limbs, abdomen, face, and airway. Although uncommon, with an estimated prevalence of roughly 1 in 50,000 people worldwide, the condition carries substantial physical, emotional, and clinical burden due to the unpredictability and potential severity of attacks.
DAWNZERA previously received approval from the U.S. Food and Drug Administration in August 2025 for preventing HAE attacks in adults and adolescents 12 years and older. Otsuka holds exclusive rights to develop and commercialize donidalorsen in Europe and the Asia-Pacific region and continues to prioritize expanding patient access.
The positive CHMP opinion represents a significant milestone in the global advancement of donidalorsen. The companies anticipate the European Commission’s decision and remain focused on addressing unmet needs and improving long-term care options for individuals living with HAE.
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