Kalvista Pharmaceuticals, Inc. has just received FDA approval for its groundbreaking oral on-demand treatment for hereditary angioedema (HAE) called Ekterly® (sebetralstat). This marks a significant development in the management of HAE, as Ekterly® is the first new on-demand HAE treatment to be approved in over a decade. The approval covers adult and pediatric patients aged 12 years and older.
The efficacy and safety of Ekterly® were established through the largest clinical trial program ever conducted in HAE, known as the Konfident clinical trial. Data from this trial, published in the New England Journal of Medicine in May 2024, revealed that Ekterly® achieved significantly faster symptom relief, reduction in attack severity, and attack resolution compared to placebo. The trial included 136 HAE patients from 66 clinical sites across 20 countries.
Furthermore, the more recent Konfident-S open-label extension trial demonstrated that Ekterly® enabled patients to treat attacks in a median of 10 minutes following onset. The data also indicated that the beginning of symptom relief occurred in a median of 1.3 hours among attacks involving the larynx, the abdomen, and for breakthrough attacks among patients receiving long-term prophylaxis. The safety profile of Ekterly® 600 mg in Konfident-S, involving a much larger number of attacks (>1700), was consistent with that observed in Konfident.
Prior to the approval of Ekterly®, all on-demand HAE treatment options approved in the U.S. required intravenous or subcutaneous administration, which presented a significant treatment burden. This approval represents a significant advancement, providing patients and physicians with a much-needed oral treatment option that offers greater independence and control over managing the condition.
In response to the approval, Kalvista Pharmaceuticals is set to launch Ekterly® in the U.S. immediately, with physicians able to begin writing prescriptions right away. The company has also established Kalvista Cares™, a comprehensive patient support program offering personalized services and resources for eligible individuals, including assistance with navigating insurance coverage, access support, and ongoing help throughout the treatment journey. Following these announcements, the company's shares moved 1.7%, and are now trading at a price of $11.98. For the full picture, make sure to review KalVista Pharmaceuticals's 8-K report.
