Prevail Therapeutics Inc., a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, 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 Neuronopathic Gaucher Disease
Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as seizures and gaze and motor abnormalities. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.