Voretigene neparvovec receives rare pediatric disease designation from FDA

The FDA offices of Orphan Products Development and Pediatric Therapeutics have designated voretigene neparvovec as a drug for a rare pediatric disease, according to a Spark Therapeutics press release.

Voretigene neparvovec, which will be marketed under the name Luxturna, could be both the first gene therapy for a genetic disease and the first pharmacologic treatment for an inherited retinal disease in the United States, according to the release. It is being reviewed for the treatment of biallelic RPE65 mutation-associated retinal dystrophy. A biologics license application for Luxturna was recently accepted by the FDA.

If the biologics license application for Luxturna is approved, Spark may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product, under the FDA’s Rare Pediatric Disease Priority Review Voucher program, the release said.