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ARTICLE | Clinical News

FDA grants Priority Review to BLA for Spark's ophthalmic gene therapy

July 21, 2017 8:34 PM UTC

Spark Therapeutics Inc. (NASDAQ:ONCE) said FDA accepted and granted Priority Review to a BLA for gene therapy Luxturna voretigene neparvovec (AAV2-hRPE65v2) to treat vision loss due to confirmed biallelic retinal pigment epithelium-specific protein 65kDa (RPE65)-mediated inherited retinal disease (IRD). Its PDUFA date is Jan. 12, 2018.

Spark said the therapy, which consists of an adeno-associated virus (AAV) encoding the RPE65 gene, could be the first gene therapy approved in the U.S., as well as the first pharmacologic treatment for an IRD. The product has rare pediatric disease designation in the U.S. for the indication...

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