Prevail Therapeutics’ (NASDAQ:PRVL) PR001 received FDA fast track designation for the treatment of neuronopathic Gaucher disease.
Gaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1 that leads to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also known as Type 2 or Type 3 Gaucher disease.
Type 2 Gaucher disease presents in infancy and leads to death by early childhood, whereas Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations.
“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,” Dr. Asa Abeliovich, Prevail’s founder and CEO, said in a statement.
“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,” he added.
Prevail plans to begin enrollment in its Phase 1/2 PROVIDE clinical trial of PR001 for the treatment of Type 2 Gaucher disease by the end of 2020.