FDA Grants Priority Review to Spark’s Gene Therapy for Retinal Disease

Spark Therapeutics’ Luxturna (voretigene neparvovec) gene therapy was granted priority review by the FDA for the one-time treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease.

According to Spark, Luxturna has the potential to be both the first pharmacologic treatment for IRD and the first gene therapy for a genetic disease in the country. The FDA assigned a PDUFA review date of Jan. 12, 2018. Inherited retinal diseases are a group of rare blinding conditions caused by one of more than 220 different genes, which can progress to total blindness.

Luxturna was evaluated in two open-label Phase I trials and a randomized, open-label Phase III trial. At one year after treatment, results showed statistically significant differences in full-field light sensitivity testing and in the ability to navigate lighted obstacles. Changes in visual acuity, a secondary endpoint, were not statistically significant.