Sumitomo Pharma Co. filed for regulatory approval in Japan Tuesday for what could become one of the country’s first commercially available treatments using induced pluripotent stem cells, targeting patients with advanced Parkinson’s disease.
The Osaka-based drugmaker’s application follows a small clinical trial involving seven patients aged 50 to 69, where researchers transplanted either 5 million or 10 million dopamine-producing cell precursors into both sides of participants’ brains. Four patients showed motor function improvements during the two-year study period, with no major adverse effects recorded.
The regulatory filing represents a test case for Japan’s accelerated approval system for regenerative medicines. Under the country’s conditional approval framework, companies can market treatments after limited trials, then must collect effectiveness data for seven years to secure final authorization.
Japan has invested more than ¥110 billion ($770 million) in regenerative medicine research since developing iPS cell technology in 2006, yet few products have reached patients. Two previous stem cell treatments approved under the conditional system have since been withdrawn due to ineffectiveness.
The treatment uses cells developed from healthy donors into precursors of dopamine-producing brain cells, which deteriorate in Parkinson’s patients. Sumitomo Pharma faces competition from companies including BlueRock Therapeutics, which is advancing a similar therapy into Phase 3 trials in the United States.
