Spark: FDA accepts BLA, grants Priority Review to voretigene neparvovec
Spark Therapeutics announced that the FDA has accepted for filing the Biologics License Application, or BLA, 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, or IRD. The investigational gene therapy, which has the proposed trade name LUXTURNA, 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. Priority Review is granted to therapeutics that would offer major advances over existing therapies or would provide a treatment where no adequate therapy exists. FDA's goal for taking an action on Priority Review applications is six months. For the review of LUXTURNA, FDA has assigned a PDUFA date of Jan. 12, 2018.