Sumitomo Pharma’s has received conditional and time-limited approval in Japan for the manufacturing and marketing authorisation of Amchepry (raguneprocel).
Raguneprocel, a stem cell-based dopamine neuron replacement therapy for treating motor symptoms in Parkinson’s disease (PD), is only available to eligible patients in the Japanese healthcare system.
The treatment involves the use of donor cells that are developed in the laboratory into dopamine-producing neurons, which are then surgically transplanted into the putamen, a region of the brain involved in movement control.
In a phase 1/ 2 clinical trial at Kyoto University Hospital, seven people with PD aged 50 to 69 were followed for 24 months after transplantation. Out of the six patients evaluated for motor outcomes, four showed potential clinical benefits when off medication and five when on medication.
After brain imagining demonstrated the transplanted cells were producing dopamine, researchers concluded the cells had survived, produced dopamine, did not form tumours and showed sign of potential clinical benefit.
Brian Fiske, chief scientist at the Michael J. Fox Foundation, said: “The limited, conditional approval of raguneprocel marks a significant scientific milestone for the Parkinson’s community.
“It reflects decades of rigorous research into regenerative approaches, and while not available in the US, it offers encouraging evidence that cell-based therapies may one day become a meaningful part of Parkinson’s treatment.”
The conditional approval lasts seven years, during which the company must collect more safety and efficacy data before the treatment can qualify for full approval.
Amchepry received the SAKIGAKE Designation for regenerative medical products in February 2017 and was designated an orphan regenerative medical product in December 2025 by the Ministry of Health, Labour and Welfare.