FDA Awards Orphan Drug Designation to Prevail's Gene Therapy for Gaucher Disease

The U.S. Food and Drug Administration (FDA) awarded Orphan Drug designation to a gene therapy under development by Prevail Therapeutics as a potential treatment for patients with Gaucher disease. The therapy was also granted Rare Pediatric Disease designation for neuronopathic Gaucher disease (nGD), the most severe form of the condition.