FDA approves Dawnzera for HAE attack prevention in patients 12+

The U.S. Food and Drug Administration has approved Dawnzera donidalorsen for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and children aged 12 and older. Dawnzera is the first RNAtargeted medicine approved for HAE and works by blocking a key protein that activates inflammatory mediators involved in swelling attacks. The self-administered therapy is given as an 80 mg dose via subcutaneous autoinjector every four weeks or every eight weeks, offering a flexible, at-home treatment option for patients with this rare, potentially life-threatening condition.

The approval is based on data from the Phase 3 OASISHAE study, which showed Dawnzera reduced the monthly HAE attack rate by about 81% compared with placebo over 24 weeks when dosed every four weeks. The benefit increased to roughly 87% when measured starting from the second dose, and the reduction in moderate-to-severe attacks was about 90% from the second dose onward. An open-label extension, OASISplus, indicated continued efficacy over longer treatment periods, including a year in which participants experienced a substantial overall decrease in attack frequency. The trial also evaluated a switch cohort...