The US Food and Drug Administration (FDA) has granted Fast Track Designation for PR001, a treatment of Parkinson’s disease. The therapy is indicated for patients with a GBA1 mutation, a group which comprises seven to 10 percent of patients with the condition.

Prevail Therapeutics, which produces the medication, says it is a potentially disease-modifying, single-dose, AAV9-based gene therapy. It can also be administered for the treatment of Gaucher disease.

"With no treatments available that modify the progressive course or the underlying disease process of Parkinson’s disease, a potential disease-modifying therapy like PR001 could significantly transform the lives of patients with this disease," said Dr Asa Abeliovich, Founder and Chief Executive Officer of Prevail.

Following the FDA’s acceptance of the company’s Investigational New Drug (IND) application in June, the Company will begin dosing patients in a Phase I/II clinical trial later this year. The trial is intended to measure safety, key biomarkers and exploratory efficacy endpoints.

-----------------------------------------------------------------------
Editor's Note:

If you have any suggestion to the content, 

please email: Julia.Zhang@ubmsinoexpo.com