Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted conditional and time-limited approval to two therapies derived from induced pluripotent stem cells (iPSCs), marking a global regulatory first for the technology. 

The approvals cover ReHeart, an iPSC-derived cardiomyocyte sheet developed by Cuorips for severe heart failure, and Amchepry, a dopaminergic neural progenitor cell therapy for Parkinson’s disease developed by Sumitomo Pharma and Racthera. Both products aim to replace damaged or lost cells using tissues generated from reprogrammed human cells. 

ReHeart is intended for patients with severe ischemic heart failure who have exhausted conventional treatment options. The therapy uses sheets of cardiomyocytes derived from donor iPS cells that are transplanted onto the surface of the heart, with the goal of supporting myocardial repair and improving cardiac function. 

Amchepry targets Parkinson’s disease, which results from the progressive loss of dopamine-producing neurons in the brain. In a small phase I/II clinical trial last year, researchers transplanted iPSC-derived dopaminergic progenitor cells into seven patients and followed them for 24 months. The study reported no serious adverse events and found that several participants experienced improvements in motor symptoms, alongside evidence of increased dopamine production in the brain. 

Japan’s approval uses the country’s conditional and time-limited authorization pathway for regenerative medicines, which allows therapies with early clinical evidence to reach patients while additional safety and efficacy data are collected after commercialization. Companies can commercialize the products for a limited period while continuing to monitor patient outcomes. 

The decision follows a February recommendation from Japan’s Pharmaceutical Affairs Council, which advised the health ministry to advance both therapies toward approval. 

The move highlights Japan’s ongoing push to accelerate regenerative medicine, an effort closely tied to the discovery of iPS cells by Shinya Yamanaka and colleagues in 2006. 

However, the clinical evidence supporting the therapies remains limited. Both treatments have so far been evaluated only in small early-stage trials involving fewer than ten patients, meaning larger controlled studies will be needed to establish their safety and effectiveness. 

Developers say the approvals will allow broader evaluation of the treatments while continuing to gather clinical evidence.