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Home » FDA Grants Priority Review to Spark’s Gene Therapy for Retinal Disease
FDA Grants Priority Review to Spark’s Gene Therapy for Retinal Disease
July 19, 2017
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.
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