Spark gene therapy wins unanimous FDA panel vote

FDA's Cellular, Tissue and Gene Therapy Advisory Committee voted 16-0 that the benefit-risk profile for Luxturna voretigene neparvovec from Spark Therapeutics Inc. (NASDAQ:ONCE) supports the gene therapy's approval to treat biallelic retinal pigment epithelium-specific protein 65kDa (RPE65) mutation-associated retinal dystrophy.

Luxturna's BLA was based on a Phase III, open-label controlled trial in 31 patients in which the AAV gene showed a statistically significant improvement on the novel endpoint of multi-luminance mobility testing (MLMT), an assessment of functional vision...