FDA backs faster development path for Parkinson’s cell therapy
Sasineprocel gets regenerative medicine advanced therapy designation
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- FDA granted RMAT designation to sasineprocel, a cell therapy for Parkinson's disease.
- This therapy uses engineered stem cells to replace lost dopaminergic neurons.
- Early trial data indicates sasineprocel is safe, improving patient function and quality of life.
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to sasineprocel (ANPD001), an experimental cell therapy to treat Parkinson’s disease.
The designation, given to promising regenerative therapies (treatments that aim to replace or restore disease-damaged tissue) that could substantially improve care for serious diseases, provides developer Aspen Neuroscience with perks such as more intensive FDA guidance.
“This significant milestone highlights the transformative nature of sasineprocel as a potentially disease-modifying therapy for patients facing a serious disease with substantial unmet medical need,” Damien McDevitt, PhD, president and CEO of Aspen, said in a company press release. “We stand with the Parkinson’s community realizing the urgency around the serious and growing unmet need in [Parkinson’s] and are unwavering in our commitment to bring sasineprocel to patients as soon as possible.”
On the fast track
The FDA previously granted sasineprocel fast track designation, a regulatory status the agency uses to speed the development of new therapies that have the potential to be transformative.
“FDA has created this collaborative regulatory framework to facilitate early and frequent interactions, align on development requirements and potentially support more efficient pathways to approval,” said Ana Sousa, Aspen’s chief regulatory officer.
Parkinson’s is marked by the loss of dopaminergic neurons, nerve cells that produce the signaling molecule dopamine. Sasineprocel works by collecting skin cells from a patient and engineering them into stem cells that can grow into dopaminergic neurons. The modified cells are then injected into the brain to restore nerve cells lost to the disease.
“Our team is working tirelessly to advance this investigational therapy designed to regenerate neural networks and restore lost neuronal function with the vision to potentially improve motor function, enhance quality of life, and reverse disease progression for patients,” Sousa said.
The FDA’s decision to grant sasineprocel RMAT designation was based primarily on data from an ongoing Phase 1/2a clinical trial, ASPIRO (NCT06344026), which is evaluating the safety of sasineprocel in people with Parkinson’s. Early data from the study have indicated that the cell therapy was generally safe and well tolerated, and that it improved daily functioning and quality of life for patients.
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