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 (HAE) causes sudden, severe swelling attacks that can block airways or distort limbs, often starting in childhood. Sebetralstat (Ekterly) is an oral drug that blocks plasma kallikrein, an enzyme that triggers the cascade of fluid leakage and swelling during HAE attacks. By stopping this process early, Ekterly may help patients control swelling episodes quickly without injections.
This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult Patients
Source: ClinicalTrials.gov