Spark Therapeutics announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have designated investigational Luxturna (voretigene neparvovec) as a drug for a rare pediatric disease. Under FDA's Rare Pediatric Disease Priority Review Voucher program, companies who receive approval for a new drug application or biologics license application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a Priority Review of a subsequent marketing application for a different product. The Priority Review voucher may be used by the company or sold to a third party.
FDA’s Office of Tissues and Advanced Therapies currently is reviewing the BLA for Luxturna for the treatment of patients with vision loss due to confirmed biallelic RPE65mutation-associated retinal dystrophy under a Priority Review designation with a Prescription Drug User Fee Act (PDUFA) action date of Jan. 12, 2018.
Luxturna has the potential to be both the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease approved in the United States. A natural history study has shown that people with this IRD eventually progress to complete blindness.