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.

New York-based Prevail said it was excited about the designation of the regulatory agency awarded to its AAV-based gene therapy PR001, which is also being developed as a potentially disease-modifying, single-dose gene therapy for Parkinson’s disease with GBA1 mutation (PD-GBA). Asa Abeliovich, founder and chief executive officer of Prevail, said the FDA-awarded designations support the company’s conviction that “new gene therapies for Gaucher disease are urgently needed — especially for the severe, neuronopathic form of the disease, for which there are no FDA-approved therapies.” Abeliovich added that the designation “underscore” the critical nature of the company’s work.

Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1. 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.

Orphan Drug Designation is granted by the FDA to drugs or biologics intended to treat a rare disease or condition, defined as one that affects fewer than 200,000 people in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity. Rare Pediatric Disease Designation is granted by the FDA in the case of serious or life-threatening diseases affecting fewer than 200,000 people in the United States, primarily those 18 years of age and younger.

In December, Prevail was cleared by the FDA for its Investigational new Drug Application for PR001 for neuronopathic Gaucher disease, also known as Type 2 or Type 3 Gaucher disease. The IND had previously been placed on clinical hold by the FDA but was removed. The planned Phase I/II clinical trial for PR001 includes a higher dose starting point than the company originally proposed. The company anticipates enrolling patients in this trial in the first half of this year. Type 2 Gaucher disease is the more severe form of nGD, which is seen in infancy and rapidly progresses into neurodegeneration leading to death in infancy or early childhood.

Prevail also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020 under the same IND.