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.
