ARTICLE | Company News
Spark hints at price for Luxturna
November 8, 2017 12:48 AM UTC
On its 3Q17 financial results call Tuesday, 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)...