Breakthrough designation for Spark's SPK-RPE65

FDA granted breakthrough therapy designation to SPK-RPE65 ( AAV2-hRPE65v2) from Spark Therapeutics LLC (Philadelphia, Pa.) for the treatment of nyctalopia, or night blindness, in patients with Leber's congenital amaurosis (LCA) due to a mutation in the retinal pigment epithelium-specific protein 65kDa (RPE65) gene. SPK-RPE65 is an adeno-associated viral (AAV) serotype 2 vector encoding the RPE65 gene.

Spark said FDA based its designation on data from two clinical trials showing that SPK-RPE65 had long-lasting effects on vision, allowing patients to perform daily activities with greater independence. The company is conducting a Phase III trial of SPK-RPE65 to treat Type 2 Leber's congenital amaurosis (LCA2), a form of retinal degeneration, and expects data in 2H15. ...