KalVista Pharmaceuticals (NASDAQ:KALV) has received marketing and manufacturing approval from Japan’s Ministry of Health, Labour and Welfare (MHLW) for EKTERLY (sebetralstat), establishing it as the first and only oral on-demand treatment for hereditary angioedema (HAE) in the country.
The approval, announced Monday, clears the novel plasma kallikrein inhibitor for use in adults and adolescents aged 12 and older to treat acute swelling attacks.
Hereditary angioedema is a rare genetic disorder characterized by sudden, painful, and potentially life-threatening swelling in various parts of the body.
Historically, on-demand treatment in Japan has relied on injectable therapies, which can be burdensome for patients to carry and administer during an attack.
The introduction of EKTERLY as an oral tablet is expected to shift the treatment paradigm by allowing patients to intervene immediately upon the onset of symptoms, reducing the risk of attack progression.
The MHLW’s decision was supported by results from the Phase 3 KONFIDENT clinical trial, the largest study of its kind in HAE history.
Published in the New England Journal of Medicine, the trial demonstrated that sebetralstat provided significantly faster symptom relief and attack resolution compared to a placebo.