FDA Grants Fast Track Designation to Autologous Investigational Cell Therapy for Parkinson Disease

ANPD001 (Aspen Neuroscience, San Diego, CA), a personalized, autologous investigational cell therapy product, has been granted Fast Track designation by the Food and Drug Administration (FDA) for investigation as a potential treatment for Parkinson disease (PD). ANPD001 is intended to replace lost dopamine neurons via the implantation of dopamine neuronal precursor cells (DANPCs), which are manufactured from induced pluripotent stem cells (iPSCs) derived from a patient’s own skin cells.

In 2022, Aspen Neuroscience launched the Trial Ready Screening Cohort study to begin enrolling participants and manufacturing individualized cells for future clinical trials. The company plans to initiate a phase 1/2a single-arm, open label dose-escalation clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ANPD001 in people with moderate-to-severe PD. It will be the first multicenter phase 1/2a trial in the United States for an autologous iPSC-derived therapy.

"We are pleased that FDA has granted Fast Track Designation, underscoring the potential of an autologous dopamine replacement therapy such as ANDP001 to serve as a meaningful treatment option," said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience. "People with Parkinson's disease have a significant unmet medical need and have been waiting for many years for more advanced treatment options."

Fast Track designation is associated with the expedited development and review of drugs designed to treat serious medical conditions and fill an unmet medical need and grants eligibility for accelerated approval and priority review.