A Parkinson’s cell therapy has gained FDA RMAT designation, giving its development programme access to closer regulatory guidance.
The US Food and Drug Administration granted the designation to sasineprocel, also known as ANPD001, Aspen Neuroscience’s lead experimental treatment for Parkinson’s disease.
Regenerative Medicine Advanced Therapy designation provides increased FDA guidance and support intended to speed up development. It may also make a treatment eligible for priority review or accelerated approval.
To qualify, a regenerative medicine therapy must be intended to treat, modify, reverse or cure a serious condition and show preliminary clinical evidence that it could address an unmet medical need.
The designation does not mean the treatment has been approved.
Damien McDevitt, president and chief executive officer of Aspen Neuroscience, said: “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.
“We stand with the Parkinson’s community realising the urgency around the serious and growing unmet need in PD and are unwavering in our commitment to bring sasineprocel to patients as soon as possible.”
Sasineprocel previously received FDA Fast Track designation, another programme intended to support the development of treatments for serious conditions with unmet medical needs.
Aspen Neuroscience said the RMAT designation was based on results from its ongoing phase 1/2a ASPIRO clinical trial.
The company said the trial had shown signs of early clinical activity and a favourable safety profile, although detailed results were not included in the announcement.
ASPIRO is an open-label, multicohort and multicentre trial, meaning researchers and participants know which treatment is being given and several groups are being studied at different clinical centres.
It is assessing the safety, tolerability and activity of surgically delivering dopamine-producing neuron precursor cells into the putamen, the part of the brain where dopamine signalling needs to be restored.
Ana Sousa, chief regulatory officer, said: “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.
“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.”
Aspen Neuroscience is developing autologous induced pluripotent stem cell therapies, which use a patient’s own cells to produce specialised cells for treatment.
Sasineprocel is a single-dose experimental therapy designed to replace dopamine-producing neurons lost through Parkinson’s disease and reconstruct neural circuits affected as the condition progresses.
A small skin punch biopsy is taken from the patient and its cells are reprogrammed into induced pluripotent stem cells. These are cells returned to an immature state so they can be developed into other specialised cell types.
The cells are then turned into precursors of dopaminergic neurons, the brain cells that produce dopamine and become damaged or lost in Parkinson’s disease.
They are delivered to the putamen using image-guided surgery.
The approach is intended to create an active cellular environment that supports the transplanted cells as they attach, survive and become integrated into the brain.
Aspen Neuroscience believes this could produce lasting benefits and potentially slow or halt the progression of Parkinson’s disease, although these benefits have not been established.
Because the therapy uses a patient’s own cells, immunosuppression, which prevents the body rejecting donor cells, is not required.
The California Institute for Regenerative Medicine, a California state agency supporting regenerative medicine, stem cell and gene therapy research, provided funding for the first two trial cohorts.
