FDA accepts NDA for sebetralstat for oral on-demand treatment of hereditary angioedema.- KalVista Pharma

If approved, sebetralstat would be the first oral, on-demand treatment for HAE in adult and pediatric patients aged 12 years and older. The FDA is not currently planning to hold an advisory committee meeting to discuss the application.

“We are thrilled with the FDA’s acceptance of our NDA for sebetralstat as it moves us one step closer to bringing a potentially transformative therapy to the HAE community,” said Ben Palleiko, Chief Executive Officer at KalVista. “We understand that people living with HAE and their families carry a tremendous burden every day as they don’t know when the next attack may occur or if the attack could cause life-threatening consequences. The compelling data included in our NDA package show that sebetralstat has the potential to significantly alter the way people treat and manage their disease. Given that it could be the first, oral on-demand treatment for HAE, we continue to receive strong support and hear a sense of urgency among healthcare providers, advocates, patients and their families for sebetralstat".

The NDA submission was supported by previously disclosed results, including data from the KONFIDENT phase III Clinical trial and ongoing KONFIDENT-S open label extension trial. Sebetralstat met the primary endpoint for its phase III trial with both 300 mg and 600 mg formulations achieving the beginning of symptom relief significantly faster than placebo (p<0.0001 for 300 mg, p="0.0013" for 600 mg) and was well-tolerated, with a safety profile similar to placebo. in konfident-s, sebetralstat has enabled patients to treat attacks early with a median time from attack onset to treatment of 9 minutes, demonstrated a consistent safety and efficacy profile with konfident, and included a median time to beginning of symptom relief for laryngeal attacks of 1.3 hours.

KalVista’s KONFIDENT-KID clinical trial, designed to evaluate the safety and efficacy of sebetralstat in a pediatric population aged 2-11 years, was initiated ahead of schedule in June 2024 and has since started dosing patients.

In addition to the NDA acceptance, KalVista recently announced that the European Medicines Agency (EMA) validated the submission of the Marketing Authorization Application (MAA) for sebetralstat. KalVista expects to file for approval in the UK, Japan, and other countries later in 2024.