for hereditary angioedema (HAE) — on-demand treatment (ages 12+)
FDA approved Ekterly (sebetralstat) on July 2, 2025 — ~2 weeks after the missed June 17 PDUFA goal date. First and only oral on-demand treatment approved for HAE attacks in adults and pediatric patients ages 12+. The KONFIDENT Phase 3 trial supported the application.
Hereditary angioedema is a rare genetic condition that causes sudden, severe, and recurrent swelling attacks throughout the body. Ekterly (sebetralstat) is an oral pill that works by blocking the plasma kallikrein enzyme, a key driver of these swelling episodes. By inhibiting this enzyme quickly, the drug could help stop an attack in its tracks, offering a convenient oral option for relief.
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
Source: ClinicalTrials.gov