The Food and Drug Administration (FDA) has granted Fast Track designation to PR001 (Prevail Therapeutics) for the treatment of Parkinson disease patients with a GBA1 mutation (PD-GBA).

PR001 is an investigational single-dose gene therapy that utilizes an AAV9 viral vector to deliver the GBA1 gene to a patient’s cells. Mutations in the GBA1 gene can lead to a deficiency of beta-glucocerebrosidase, leading to an accumulation of glycolipids and lysosomal dysfunction in CNS cells. The Company believes that this is what leads to the inflammation and neurodegeneration observed in patients with PD-GBA.

A phase 1/2 clinical trial is being planned for the second half of 2019 to investigate the safety and tolerability of PR001 along with key biomarkers and exploratory efficacy end points in patients with PD-GBA. PR001 is also being developed for the treatment of neuronopathic Gaucher disease.

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“We are pleased that the FDA has granted Fast Track designation for PR001, which underscores the unmet need of patients with Parkinson disease with a GBA1 mutation, a chronic and progressive neurodegenerative disorder that comprises 7% to 10% of the total Parkinson disease population worldwide,” said Asa Abeliovich, MD, PhD, Founder and Chief Executive Officer of Prevail. 

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