Ionis Pharmaceuticals Receives Approval for DAWNZERA™, the First RNA-Targeted Prophylactic Treatment for Hereditary Angioedema
Friday, August 22, 2025
Ionis Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved DAWNZERA™ (donidalorsen) for the prevention of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and above.
DAWNZERA is the first and only RNA-targeted therapy approved for HAE, designed to block plasma prekallikrein (PKK), a protein involved in triggering acute swelling episodes.
HAE is a rare, life-threatening genetic condition that causes repeated episodes of severe swelling in areas such as the face, throat, abdomen, hands and feet. It is estimated to affect around 7,000 people in the United States.
DAWNZERA is designed to block plasma prekallikrein (PKK), a protein that activates inflammatory mediators linked to acute HAE attacks. The medicine is administered through a subcutaneous autoinjector at a dose of 80 mg, either once every four weeks (Q4W) or once every eight weeks (Q8W).
The approval was supported by results from the Phase 3 OASIS-HAE study, where DAWNZERA given every four weeks reduced the monthly HAE attack rate by 81% compared with placebo over 24 weeks. When measured from the second dose, the reduction increased to 87%. The treatment also lowered the rate of moderate-to-severe attacks by around 90%.
Across clinical trials, DAWNZERA demonstrated a favourable safety profile. The most common side effects were injection site reactions, upper respiratory infections, urinary tract infections and abdominal discomfort.
The treatment will become available in the United States in the coming days.
Source: ionis.com
