Japan-based Sumitomo Pharma has applied for marketing authorization from Japanese regulators for what could become the world's first Parkinson's disease treatment utilizing induced pluripotent stem cells (iPSCs).
Raguneprocel consists of allogeneic iPSC-derived dopaminergic neural progenitor cells designed to improve of motor functions during the off-time period of patients with advanced Parkinson’s disease. The cells are bilaterally transplanted into the brain using a neurosurgical navigation system.
The submission — which has priority review from Japan’s Ministry of Health, Labour and Welfare — is based on the data from an investigator-initiated phase 1/2 trial conducted by Kyoto University Hospital. Among six patients subjected to efficacy evaluation, four showed improvements in the Movement Disorder Society Unified Parkinson’s Disease Rating Scale part III OFF score, and five showed improvements in the ON scores.
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