CAMBRIDGE, Mass.– KalVista Pharmaceuticals has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for sebetralstat, a new oral therapy for hereditary angioedema (HAE) attacks. If approved by the European Commission—expected by early October—sebetralstat would become the first and only oral on-demand treatment for HAE available in the European Union.
Sebetralstat, an oral plasma kallikrein inhibitor, is intended for the treatment of acute HAE attacks in adults and adolescents aged 12 and older. The CHMP’s recommendation is based on data from the Phase 3 KONFIDENT trial, the largest HAE clinical trial to date. Results, published in The New England Journal of Medicine in May 2024, showed that sebetralstat significantly reduced symptom severity and provided faster relief and attack resolution compared to placebo, while maintaining a safety profile similar to placebo.
“This positive CHMP opinion is an important step forward for people living with HAE in Europe, where there remains a high need for effective, easy-to-administer on-demand treatments,” said KalVista CEO Ben Palleiko. “Sebetralstat is expected to become the first and only oral on-demand treatment for HAE in Europe, enabling patients to treat attacks quickly and independently, wherever they occur.”
Current on-demand HAE treatments in Europe rely on parenteral (injection-based) administration. Experts say an oral option like sebetralstat could mark a major shift in treatment.
“Access to on-demand treatments that offer easy self-administration and rapid symptom relief is critical for patients living with HAE,” said Dr. Emel Aygören-Pürsün of University Hospital Frankfurt. “This positive opinion is an essential move toward a long-awaited oral option that aligns with treatment guidelines and could help patients lead more normal lives.”
Sebetralstat, marketed in the U.S. and U.K. under the brand name EKTERLY®, is also under regulatory review in Japan and several other territories.
