iRegene Therapeutics has received regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its lead product, NouvNeu001, aimed at treating Parkinson’s disease.
This marks the first instance of an allogeneic induced pluripotent stem cell (iPSC)-derived cell therapy receiving both fast track and RMAT designations from the FDA, with the former awarded in August 2025.
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The FDA introduced the RMAT designation, a regulatory pathway, under the 21st Century Cures Act. It is intended to expedite the development and evaluation of regenerative medicine therapies intended for serious or life-threatening diseases.
Therapies receiving this designation may have earlier engagement with the agency and could qualify for accelerated approval and priority review.
NouvNeu001 is designed to address neuronal loss and support the restoration of natural dopamine production by targeting neuronal degeneration at its root.
Phase I data showed significant clinical efficacy, including improvements in movement disorder society-unified Parkinson’s disease rating scale (MDS-UPDRS) Part III motor scores.
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By GlobalDataiRegene chief medical officer Dr Meng Cai said: “The RMAT designation is a pivotal regulatory advancement for NouvNeu001 and a strong endorsement of its clinical value.
“This designation reflects the FDA’s recognition of NouvNeu001’s potential to address a serious unmet medical need in Parkinson’s disease and provides a structured framework for deep, efficient collaboration with the agency.
“We are committed to leveraging this opportunity to accelerate our global clinical development programme and bring this transformative therapy to patients as early as possible.”
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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