Prevail Therapeutics Receives U.S. FDA Fast Track Designation for PR001 for the Treatment of Neuronopathic Gaucher Disease

Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s experimental gene therapy program, PR001, for the treatment of neuronopathic Gaucher disease (nGD). The Company expects to initiate enrollment of the PROVIDE Phase 1/2 clinical trial of PR001 for Type 2 neuronopathic Gaucher disease patients in the second half of 2020.

“FDA Fast Track designation for PR001 for the treatment of neuronopathic Gaucher disease underscores the significant unmet medical need for this devastating condition, for which there are no currently approved therapies,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. “Type 2 Gaucher disease involves rapidly progressive neurodegeneration leading to death in infancy or early childhood, and Type 3 Gaucher disease is associated with significant neurological manifestations including seizures and motor abnormalities. We believe PR001 has the potential to serve as a much-needed therapeutic option for these patients as enzyme replacement therapies approved for Type 1 Gaucher disease cannot cross the blood brain barrier to address neurological symptoms.”

The FDA previously granted PR001 Rare Pediatric Disease Designation for the treatment of nGD, and Orphan Drug Designation for the treatment of patients with Gaucher disease. In addition, the FDA has granted Fast Track designation for PR001 for the treatment of Parkinson’s disease with GBA1 mutations.

About Fast Track Designation
The FDA’s Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

To Read the Complete Article at GlobeNewswire, Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Stock image by @SergeyNivens from Depositphotos