FX.co ★ KalVista Announces Orphan Drug Designation And NDA Submission For Sebetralstat In Japan

KalVista Pharmaceuticals, Inc. (KALV) revealed on Tuesday that Japan's Ministry of Health, Labour and Welfare (MHLW) has conferred Orphan Drug Designation upon sebetralstat. Additionally, the company has submitted a New Drug Application (NDA) for sebetralstat to the Ministry.

Pending approval, sebetralstat would be groundbreaking as the first oral treatment available in Japan for on-demand use against hereditary angioedema (HAE) attacks in both adults and adolescents aged 12 and above. This investigational therapy functions as a plasma kallikrein inhibitor.

The NDA submission is backed by previously disclosed results, notably from the KONFIDENT Phase 3 clinical trial and the ongoing KONFIDENT-S open-label extension study.

The KONFIDENT Phase 3 clinical trial involved a randomized, double-blind, three-way crossover design aimed at assessing the safety and efficacy of doses of 300 mg and 600 mg of sebetralstat, compared to a placebo, for on-demand treatment of HAE in patients aged 12 years and older.

In parallel, the KONFIDENT-S extension trial is an open-label study incorporating numerous real-world scenarios to evaluate the long-term safety and efficacy of sebetralstat for managing HAE attacks in both adult and pediatric patients with HAE Type I or Type II, aged 12 years and older.