FDA advisory committee recommends Luxturna (voretigene neparvovec),gene therapy, to treat vision loss due to biallelic RPE65-mediated inherited retinal disease.- Spark Therapeutics

Spark Therapeutics has announced that the FDA Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of Luxturna (voretigene neparvovec), an investigational, potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).

The advisory committee�s recommendation is based on Luxturna�s clinical development program, which includes the first completed randomized, controlled Phase III gene therapy clinical trial ever conducted for a genetic disease. In the original Phase III intervention group, participants aged four to 44 years on average maintained the functional vision and visual function improvements demonstrated 30 days after Luxturna administration through their last annual follow-up visit, as measured by bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing. Data from a cohort of the Phase 1 clinical trial, in which investigational Luxturna was administered to the contralateral, or second previously uninjected eye, showed similarly maintained mean improvements. As part of the Biologics License Application (BLA) to FDA, Spark also submitted the results of two Phase 1 clinical trials, a natural history study and a MLMT validation study. Today�s advisory committee vote is non-binding, but FDA will take its recommendation into consideration when reviewing the BLA for Luxturna.

Luxturna is under Priority Review with FDA, with an assigned Prescription Drug User Fee Act (PDUFA) date of 12 January 2018. Luxturna has received orphan drug, breakthrough therapy and rare pediatric disease designations from FDA. In August 2017, Spark Therapeutics� Marketing Authorization Application (MAA) for Luxturna was validated by European Medicines Agency (EMA). Luxturna also has received orphan product designations from EMA.

Comment: There currently are no pharmacologic treatment options for people living with RPE65-mediated IRD, who in most cases progress to complete blindness.