KalVista Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, as the first and only oral on-demand treatment for acute hereditary angioedema (HAE) attacks in patients aged 12 and older.
“This FDA approval is a defining moment for people living with HAE,” said KalVista CEO Ben Palleiko. He emphasized that EKTERLY enables patients to treat attacks as soon as symptoms start, offering convenience and independence that previous injectable treatments could not.
Currently, on-demand therapies in the U.S. require intravenous or subcutaneous administration, posing challenges for timely treatment. EKTERLY’s oral formulation represents a significant advance in patient care by simplifying administration and improving access to immediate relief.
Dr. Marc A. Riedl of the U.S. Hereditary Angioedema Association Center at UC San Diego, who participated in the phase 3 KONFIDENT trial, highlighted the importance of an oral option. “Having an oral treatment empowers patients to address attacks early, which aligns with clinical guidelines and can reduce disease burden,” he said.
EKTERLY’s approval is supported by data from the KONFIDENT phase 3 trial, the largest clinical study in HAE to date, involving 136 patients across 20 countries. Results published in the New England Journal of Medicine in May 2024 showed EKTERLY provided faster symptom relief, reduced attack severity, and had a safety profile comparable to placebo. An ongoing open-label extension study, KONFIDENT-S, further confirmed the drug’s effectiveness in real-world use.
KalVista will launch EKTERLY immediately in the U.S., with prescriptions available now. To support patients, the company has introduced KalVista Cares, a program offering assistance with insurance coverage, access support, and ongoing patient resources.
This approval marks a major milestone in HAE treatment, offering new hope for patients seeking rapid, convenient relief from unpredictable and potentially life-threatening attacks.