Spark hints at price for luxturna

On its 3Q17 financial results call, Spark Therapeutics Inc. (NASDAQ:ONCE) said its economic modeling for gene therapy Luxturna voretigene neparvovec valued the one-time treatment at over $1 million.

Luxturna is an adeno-associated virus (AAV) encoding the retinal pigment epithelium-specific protein 65kDA (RPE65) gene that is under FDA review to treat a biallelic RPE65 mutation-associated retinal dystrophy, an inherited disorder that leads to blindness. The therapy, which has a Jan. 12 PDUFA date, received unanimous support from an FDA panel last month (see BioCentury, Oct. 20)...