Prevail Earns Orphan Drug, Rare Pediatric Disease Designations for Gene Therapy

The FDA granted Prevail Therapeutics’ investigational gene therapy PR001 orphan drug and rare pediatric disease designations for treating Gaucher disease patients.

The New York City biotech company’s gene therapy received orphan drug designation for treating patients with the rare inherited metabolic disorder and a rare pediatric disease designation for the condition’s most severe form, neuronopathic Gaucher disease.

The gene therapy is also being developed as a single-dose gene therapy for Parkinson’s disease with GBA1 mutation.