Japanese Regulatory Panel Recommends Advancing World’s First iPSC-Based Therapies

Japan’s Pharmaceutical Affairs Council, an advisory body within the country’s Ministry of Health, Labor and Welfare (MHLW), has recommended the conditional and time-limited marketing approval of two induced pluripotent stem cell (iPSC)-based therapies. If formally approved by MHLW, the decision would mark the first regulatory approval of iPSC-derived therapeutic products anywhere in the world.

The first therapy, allogeneic iPSC-derived dopaminergic neural progenitor cells (Amchepry), developed by Sumitomo Pharma Co. and Racthera Inc., is designed for the treatment of Parkinson’s disease. The product consists of differentiated neural progenitor cells derived from donor iPSCs intended to restore dopaminergic signaling.

The second therapy, ReHeart, developed by Cuorips Corporation, is an allogeneic iPSC-derived cardiomyocyte patch designed to treat severe heart failure due to ischemic cardiomyopathy. The therapy involves transplantation of cardiomyocyte sheets derived from iPSCs to support myocardial repair.

In a Feb. 20 press conference, Minister of Health, Labor and Welfare Kenichiro Ueno stated that formal approval is expected as early as March. He also emphasized that the authorization would be conditional and time-limited, with continued verification of efficacy required following commercialization.