FDA grants advanced therapy status to Parkinson’s cell treatment
iRegene's NouvNeu001 given RMAT, fast track designations
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- FDA granted advanced therapy status to Parkinson's cell therapy NouvNeu001.
- The treatment replaces lost dopamine neurons to improve motor symptoms.
- Early trials show safety and significant motor function improvements.
The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) status to NouvNeu001, iRegene Therapeutics’ cell therapy for Parkinson’s disease.
The FDA based its decision on data from a Phase 1/2 trial (NCT06167681) in China, which showed that the treatment eased motor symptoms in Parkinson’s patients. The company began a Phase 1 trial (NCT07102342) in the U.S. last year.
RMAT designation aims to accelerate the development of regenerative therapies with the potential to address unmet medical needs in serious conditions. It provides companies with more interactions with the FDA during product development, including help with clinical trial design and manufacturing strategy, and potential eligibility for accelerated and priority review.
“This designation reflects the FDA’s recognition of NouvNeu001’s potential to address a serious unmet medical need in Parkinson’s disease,” Meng Cai, iRegene’s chief medical officer, said in a company press release. “We are committed to leveraging this opportunity to accelerate our global clinical development program and bring this transformative therapy to patients as early as possible.”
Delivering nerve cell precursors to brain
Parkinson’s disease is caused by the loss of dopaminergic neurons, the nerve cells that produce dopamine, a brain signaling molecule essential for motor control. The loss of these neurons impairs dopamine signaling, leading to Parkinson’s symptoms.
NouvNeu001 is composed of human dopaminergic neuron precursors (DNP), cells capable of differentiating into dopaminergic neurons. These cells are derived from donor-induced pluripotent stem cells, which are generated from mature cells and can differentiate into different cell types, including neurons.
DNPs are delivered directly into the striatum, a brain region affected in Parkinson’s, via a guided intracerebral injection. Following the transplant, DNPs are expected to differentiate into new dopaminergic neurons, form connections with existing neurons, and enhance dopamine release.
The two clinical trials are evaluating the safety, tolerability, and preliminary efficacy of a single injection.
Results from the Phase 1 part of the trial in China demonstrated that the treatment was generally safe and well tolerated for up to 15 months after transplantation, without the use of immunosuppressants after the sixth month. PET imaging revealed the long-term survival and differentiation of the transplanted cells.
Treated patients demonstrated significant improvements in motor function, as measured by the MDS-UPDRS Part III, which assesses motor symptoms. Twelve months after transplantation with the lower dose, mean scores decreased by 30.6 points during off time (a 52.82% improvement from baseline) and by 12.9 points during on time (a 54.67% improvement).
Off time refers to periods when the effects of Parkinson’s medication have worn off, and symptoms worsen. On time denotes the periods during which the medication is effective.
Comparable improvements were seen at the higher dose level, with reductions of 23.3 points in off time and 9.67 points in on time nine months after transplantation. These improvements continued for 15 months, suggesting durable therapeutic benefit.
NouvNeu001 was also granted FDA fast track designation, which aims to accelerate the development and review of therapy candidates designed to treat serious conditions and that may offer significant benefits over approved treatments.
