US FDA Approves EKTERLY® as First Oral On-Demand Treatment for HAE
KalVista Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved EKTERLY® (sebetralstat), a new oral plasma kallikrein inhibitor, for the treatment of acute attacks of hereditary angioedema (HAE) in patients aged 12 years and older. EKTERLY is now the first and only oral on-demand treatment available for HAE.
Until now, all approved on-demand treatments for HAE in the U.S. involved either intravenous or subcutaneous injections, which can be burdensome and may delay treatment.
EKTERLY provides a new oral alternative, offering patients the opportunity to treat symptoms as soon as they begin, regardless of location.
The approval is based on results from the KONFIDENT phase 3 trial, the largest clinical trial conducted in HAE to date. The study included 136 patients across 66 sites in 20 countries.
Findings published in the New England Journal of Medicine (May 2024) showed that EKTERLY led to quicker relief of symptoms, reduced severity of attacks, and faster resolution compared to placebo. The treatment was also well tolerated and had a safety profile similar to placebo.
Further support came from the ongoing KONFIDENT-S open-label extension trial, which has tracked over 1,700 HAE attacks. Results showed that patients typically took the treatment within 10 minutes of an attack starting.
Median time to symptom relief was 1.3 hours for attacks affecting areas such as the larynx and abdomen, and also for breakthrough attacks in those using long-term preventative therapies.
KalVista will begin the U.S. launch of EKTERLY immediately, and prescriptions can now be issued. The company has also introduced KalVista Cares™, a patient support programme aimed at helping eligible individuals with insurance navigation, treatment access, and ongoing support.
