Japan gave the green light to one of the most promising medical advances in the fight against Parkinson’s. Pharmaceutical companies Sumitomo Pharma and Racthera announced that the country conditionally approved the marketing of Amchepry® (raguneprocel), the first regenerative medicine treatment derived from induced pluripotent stem (iPS) cells designed to improve motor symptoms in patients with this neurodegenerative disease.
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The authorization represents a historic step in the development of cell therapies applied to the treatment of Parkinson’s. According to the companies’ announcement, the drug is aimed at patients whose motor symptoms do not respond adequately to existing drug therapies, including those containing levodopa, the drug most commonly used to control the disease.
The new product is based on a radically different approach to conventional treatments. Instead of simply relieving symptoms, it seeks to restore damaged neuronal function by implanting cells capable of becoming dopamine-producing neurons.
This disease has no cure, but it can be treated with medications. Photo:iStock
A stem cell-based therapy
Amchepry® contains dopaminergic neural progenitor cells, produced from allogeneic iPS cells. These cells have not yet fully differentiated into neurons, but they have the potential to become dopaminergic neurons, responsible for producing dopamine.
Dopamine is a neurotransmitter essential for the control of movement. In patients with Parkinson’s, the neurons that produce it progressively deteriorate, causing characteristic motor symptoms such as tremors, muscle stiffness, and difficulty moving.
The treatment uses non-frozen cells obtained through the differentiation of iPS cells. These cells are transplanted with the aim of replacing or supporting damaged neurons, helping to restore dopamine production in the brain.
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According to the companies, it is the first approved iPS cell-derived regenerative medicine product in the world, marking a milestone for this biomedical technology.
The approval is based on data from a clinical study initiated by researchers at Kyoto University Hospital. The results of this phase I/II clinical trial were published in April 2025 in the scientific journal Nature.
The new drug uses dopaminergic progenitor cells derived from iPS cells. Photo:iStock
Based on these results, Sumitomo Pharma submitted on August 5, 2025 the application for authorization to manufacture and market the treatment, which has now received regulatory approval under a conditional and time-limited approval scheme.
This type of authorization allows treatment to be introduced while additional clinical data continues to be collected. To obtain full approval, the pharmaceutical company must conduct post-marketing clinical studies, as well as safety and efficacy monitoring in patients.
How the treatment will occur
Following its inclusion in the Japanese national health insurance pricing system, Sumitomo Pharma will be responsible for marketing the drug.
Manufacturing will be carried out by S-Racmo, a contract development and manufacturing organization specializing in cellular and regenerative therapies.
The product will be produced at SMaRT, a facility located in Suita, Osaka, considered the world’s first plant dedicated to large-scale commercial manufacturing of regenerative medicine products derived from allogeneic iPS cells.
The cells used in the treatment come from an iPS cell bank provided by the CiRA Foundation, using differentiation and manufacturing technologies developed by researchers at Kyoto University and other collaborators.
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In addition, one of the steps in the production process uses a proprietary cell purification technology developed by the KAN research institute—currently integrated into the research laboratories of Eisai Co., Ltd.—that allows the dopaminergic progenitor cells necessary for the therapy to be more precisely isolated.
The treatment had previously received Sakigake designation for regenerative medicine products in February 2017 by Japan’s Ministry of Health, Labor and Welfare, a category that seeks to accelerate the development of innovative therapies.
Amchepry® therapy introduces iPS-derived cells Photo:Stock
Later, in December 2025, it was also designated as an orphan product of regenerative medicine, which is awarded to therapies aimed at diseases with unmet medical needs.
The development of the drug has been supported by the Japanese government through programs funded by the Japan Agency for Medical Research and Development (AMED). These include initiatives aimed at the realization of regenerative medicine, the practical application of these therapies and the development of key technologies for their evaluation.
The companies involved indicated that the objective of the project is to offer patients with Parkinson’s a therapeutic alternative different from conventional pharmacotherapies.
EDWIN CAICEDO
Environment and Health Journalist
@CaicedoUcros
