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 inherited condition that causes sudden, repeated attacks of severe swelling, often starting in childhood. Ekterly is an oral medicine that blocks plasma kallikrein, an enzyme that drives the swelling-causing signal during an attack. By blocking that enzyme, the drug may calm the swelling cascade quickly, giving people a pill they could take at the first sign of an attack.
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