Press release
KalVista Announces Orphan Drug Designation and NDA Submission for Sebetralstat in Japan for Hereditary Angioedema
–Orphan drug designation paves the way for potential of sebetralstat to be first oral on-demand treatment for HAE in Japan– – Submission advances Company’s
About this update from Kalvista Pharmaceuticals, Inc.
[{"type":"text","content":"\n–Orphan drug designation paves the way for potential of sebetralstat to be first oral on-demand treatment for HAE in Japan–\n\n\n– Submission advances Company’s strategic plan to address unmet needs in HAE on global scale–\n\n\n CAMBRIDGE, Mass. & SALISBURY, England--(BUSINESS WIRE)--\nKalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted sebetralstat Orphan Drug Designation. The Company has also submitted a New Drug Application (NDA) for sebetralstat to the Agency. If approved, sebetralstat, a novel, investigational oral plasma kallikrein inhibitor for the on-demand treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older, would be the first oral on-demand treatment for HAE in Japan.\n\n\n“The submission of our NDA for sebetralstat in Japan represents another key step toward our efforts to make this important new treatment available to as many people living with HAE as possible,” said Ben Palleiko, CEO of KalVista. “The Orphan Drug Designation not only acknowledges the critical need for new, effective treatments for HAE in Japan but underscores the potential of sebetralstat to provide meaningful relief for people who have faced ongoing challenges with existing options. We are proud to be at the forefront of advancing care for the HAE community.”\n\n\nThe NDA submission is supported by previously disclosed results, including data from the KONFIDENT phase 3 clinical trial and ongoing KONFIDENT-S open-label extension trial.\n\n\nAbout the KONFIDENT Phase 3 Trial\n\n\nThe KONFIDENT phase 3 clinical trial was a randomized, double-blind, 3-way crossover trial evaluating the safety and efficacy of sebetralstat 300 mg and 600 mg versus placebo for the on-demand treatment of HAE in adult and pediatric patients aged 12 years and older. The trial randomized 136 HAE patients from 66 clinical sites across 20 countries, making it the largest clinical trial ever conducted in HAE. In the trial, participants treated each eligible attack with up to two doses of study drug and treated up to three attacks over the course of the study. The trial included type 1 and type 2 HAE patients who had at least two documented HAE attacks 90 days prior to randomization and also included patients receiving long-term prophylaxis.\n\n\nAbou...