Japanese regulatory panel recommends approval of Amchepry, world's first iPSC-derived therapy

Sumitomo Pharma/Amchepry (Raguneprocel)

Japan's Pharmaceutical Affairs Council recommended approval of Sumitomo Pharma's Amchepry (raguneprocel), the world's first commercial approval of an iPSC-derived therapy. The recommendation followed a phase I/II trial showing safety and motor improvements in seven Parkinson's patients.

• Conditional and time-limited approval; data collection required over seven years for full approval
• Phase I/II trial: seven patients aged 50-69, followed 24 months, four showed motor benefits off-medication
• Brain imaging confirmed cell survival, dopamine production, and no tumour formation
• Treatment uses donor-derived iPSC-derived dopaminergic neuronal progenitors surgically implanted in the brain