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FDA Advisory Committee recommends approval of Luxturna gene therapy

13 October 2017

Spark Therapeutics, the gene therapy company, announced today that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended 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).

“Today’s unanimous advisory committee vote recommending the approval of Luxturna moves us closer to bringing this investigational adeno-associated viral (AAV) vector gene therapy to patients with vision loss due to confirmed biallelic RPE65-mediated IRD,” said Katherine A. High, M.D., president and head of Research and Development at Spark Therapeutics. “The clinical program for Luxturna includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test score change and full-field light sensitivity threshold testing, with observation ongoing. We look forward to continuing to work with FDA as it completes its review of Luxturna.”

The advisory committee’s recommendation is based on Luxturna’s clinical development program, which includes the first completed randomized, controlled Phase 3 gene therapy clinical trial ever conducted for a genetic disease. In the original Phase 3 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.

“There currently are no pharmacologic treatment options for people living with RPE65-mediated IRD, who in most cases progress to complete blindness,” said Principal Investigator Albert M. Maguire, M.D., professor of ophthalmology at the Scheie Eye Institute at the University of Pennsylvania’s Perelman School of Medicine and attending physician in the Division of Pediatric Ophthalmology at Children's Hospital of Philadelphia. “As a practicing physician who often speaks with patients and families living with IRDs, these conversations have been, up to now, frustrating in that there has been nothing to offer. Today’s advisory committee vote is an important step closer to the day that discussion can include potentially treating the blindness caused by their IRD.”

Learn more about this story on the Spark Therapeutics website by following the link below:


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