Ruijian Pharmaceuticals Receives FDA Approval to Initiate Global Phase I Clinical Trial for NouvNeu004 in Multiple System Atrophy

Recently, the U.S. FDA granted iRegene's NouvNeu004 injection "Special Exemption" status and further approved the international Phase 1 clinical trial of this product for Multiple System Atrophy (MSA).

Multiple System Atrophy (MSA) is a rapidly progressing adult neurodegenerative disease. The pathological core of MSA lies in the abnormal aggregation of α-synuclein in oligodendrocytes, leading to myelin damage and nerve conduction disorders, which gradually disrupt multiple critical brain regions. This mechanism is fundamentally different from neurodegenerative diseases such as Parkinson's disease. As a result, conventional neurological treatment strategies are often minimally effective for MSA, with patients exhibiting complex clinical manifestations and extremely high diagnostic and therapeutic challenges. MSA progresses rapidly, and most patients face life-threatening conditions within 6 to 10 years after diagnosis.

NouvNeu004 Injection adopts an innovative "neurotrophic + neural reconstruction" combination treatment strategy, with a compound mechanism of action including:The injection provides nutritional support to endangered cells in the lesion area, preventing further cell death; meanwhile, it induces differentiation into nerve cells through microenvironment modulation at multiple lesion sites, achieving systematic neural repair and functional reconstruction.

According to the press release from iRegene, two of its iPSC-derived universal cell therapies — NouvNeu001 for Parkinson's disease and NouvNeu004 for multiple system atrophy — have both been granted "Special Exemption" status by the FDA. Additionally, both therapies have achieved dual submissions and approvals in China and the U.S. NouvNeu001 was approved by the FDA in June 2024 to initiate an international multicenter Phase 1 clinical trial in the United States and other countries. In August 2025, NouvNeu001 was also granted Fast Track Designation (FTD) by the FDA.

References:
[1]FDA Grants "Special Exemption" Again, World's First Cell Therapy Product for Multiple System Atrophy Approved for International Clinical Trials. From https://mp.weixin.qq.com/s/HlTEAgCnK0S7qCzJJQvUlQ

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