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.
Crucially, the safety profile of the drug was found to be comparable to the placebo group, with no serious adverse events reported during the study.
KalVista will partner with Kaken Pharmaceutical Co., Ltd. for the commercial rollout in Japan.
While the approval is immediate, the formal launch is expected shortly after EKTERLY is listed on the Japanese National Health Insurance (NHI) System.
The partnership agreement, established earlier this year, grants Kaken exclusive commercialization rights while KalVista retains manufacturing responsibilities.
This regulatory win in Japan marks the seventh major global market approval for EKTERLY since July 2025, following successful submissions in the United States, European Union, and United Kingdom.
The rapid global expansion highlights a significant period of growth for KalVista, which received Orphan Drug designation for the therapy in Japan in late 2024.