What's Happening?
Ionis Pharmaceuticals, Inc. and Otsuka Pharmaceutical Co., Ltd. have announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has given a positive opinion
on DAWNZERA™ (donidalorsen) for the prevention of recurrent hereditary angioedema (HAE) attacks in individuals aged 12 and older. This recommendation is based on clinical evidence from the Phase 3 OASIS-HAE and OASISplus studies, which demonstrated significant reductions in HAE attack rates. The decision now awaits approval from the European Commission, expected in the first quarter of 2026. DAWNZERA was previously approved by the U.S. Food and Drug Administration in August 2025 for similar use.
Why It's Important?
The positive CHMP opinion is a significant step towards expanding treatment options for HAE, a rare genetic condition characterized by severe swelling attacks. If approved by the European Commission, DAWNZERA could provide a new preventive treatment for patients in Europe, potentially improving quality of life and reducing healthcare burdens associated with emergency treatments for HAE attacks. The approval would also mark a commercial expansion for Ionis and Otsuka, enhancing their presence in the European pharmaceutical market.
What's Next?
The European Commission's decision on DAWNZERA's approval is anticipated in early 2026. If approved, Otsuka will have exclusive rights to distribute the drug across Europe and the Asia Pacific region. This could lead to increased collaboration between healthcare providers and the pharmaceutical companies to ensure widespread access to the treatment. Additionally, the approval could prompt further research and development in RNA-targeted therapies for other rare diseases.
Beyond the Headlines
The development of DAWNZERA highlights the growing importance of RNA-targeted therapies in treating genetic conditions. This advancement may encourage further investment in similar technologies, potentially leading to breakthroughs in other areas of unmet medical need. The ethical considerations of expanding access to such treatments globally, especially in regions with limited healthcare infrastructure, may also become a topic of discussion.
