Spark Therapeutics receives Rare Pediatric Disease designation for LUXTURA(TM)
Spark Therapeutics announced the FDA has designated investigational LUXTURNATM (voretigene neparvovec) as a drug for a rare pediatric disease. Under FDA's Rare Pediatric Disease Priority Review Voucher program. LUXTURNA has the potential to be both the first pharmacologic treatment for IRD. July 20, 2017