Posted by Michael Wonder on 18 Jul 2017
Spark Therapeutics’ biologics license application for investigational voretigene neparvovec accepted for filing by FDA
17 July 2017 - FDA grants priority review with PDUFA date of 12 January 2018.
Spark Therapeutics announced today that the U.S. FDA has accepted for filing the biologics license application and granted priority review for voretigene neparvovec, an investigational, potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).
The investigational gene therapy, which has the proposed trade name Luxturna (voretigene neparvovec), has the potential to be both the first pharmacologic treatment for IRD and the first gene therapy for a genetic disease in the United States.
