FDA fast tracks Prevail’s Parkinson’s gene therapy

The FDA has fast tracked a gene therapy for a certain form of Parkinson’s Disease from US biotech Prevail Therapeutics.

New York-based Prevail is a biotech developing adeno associated virus (AAV)-based gene therapies for patients with neurodegenerative disorders.

Prevail said the FDA has granted Fast Track designation for its lead gene therapy, PR001 for treatment of Parkinson’s disease patients with a GBA1 mutation (PD-GBA).

The company described PR001 as a potentially disease-modifying, single-dose gene therapy, which is also being developed for neuronopathic Gaucher disease.

A phase 1/2 clinical trial of PR001 is due to begin dosing patients in the second half of this year.

The trial will investigate the safety and tolerability of PR001, and will measure biomarkers and exploratory efficacy endpoints in patients with PD-GBA.

Fast Track designation means the FDA will expedite review and development of products aimed at serious conditions, where there is a need for new therapies.

It allows for early and frequent dialogue with the FDA through the drug development and review process.

An accelerated approval based on mid-stage data is possible, as is a rolling review of clinical evidence to speed up the process.

Fast Track products may be eligible for a faster six-month Priority Review if clinical data is sufficient at the time of filing.

PD-GBA affects 7% to 10% of the total Parkinson’s disease population worldwide and an estimated 90,000 individuals in the US alone.

GBA1 encodes the lysosomal enzyme, beta-glucocerebrosidase, or GCase. Mutations in the GBA1 gene lead to a deficiency of GCase, resulting in lysosomal dysfunction in CNS cells, which Prevail believes leads to the inflammation and neurodegeneration present in PD-GBA.

GBA1 mutations impact the risk of developing Parkinson’s disease as well as many other aspects of the disease course, including the severity, age of onset and rate of progression of disease and the likelihood of dementia.

There are no treatments available that modify the progressive course or the underlying disease process of Parkinson’s disease.

In a separate development Seelos Therapeutics licensed gene therapy product SLS-004 from Duke University to treat patients with Parkinson’s Disease.

The company has two other products in its pipeline for treating Parkinson’s Disease – SLS-007 and SLS-006.

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