FDA Committee Recommends Approval of Spark’s Eye-Disease Gene Therapy

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The committee has voted unanimously to approve Spark Therapeutics’ gene therapy candidate, Luxturna (voretigene neparvovec), for treating a genetically inherited blindness.

On Oct. 12, 2017, Spark Therapeutics, a fully integrated gene therapy company, announced that FDA’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended the approval of Luxturna (voretigene neparvovec), an investigational, potential one-time gene therapy, for treating vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).

The committee’s recommendation is based on the therapy’s clinical development program, which includes a Phase III gene therapy clinical trial for a genetic disease, the first of its kind, according to the company. The committee’s approval vote is non-binding, but FDA will take its recommendation into consideration when reviewing the biologics license application for Luxturna.

The gene therapy is under priority review with FDA, which has assigned a Prescription Drug User Fee Act date of Jan. 12, 2018. The therapy has received orphan drug, breakthrough therapy, and rare pediatric disease designations from FDA. In August 2017, the European Medicines Agency (EMA) validated the company’s marketing authorization application for the therapy, which also received orphan product designations from EMA.

“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, MD, president and head of Research and Development, Spark Therapeutics, in a company press release. “The clinical program for [the therapy] includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing, with observation ongoing. We look forward to continuing to work with FDA as it completes its review of 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, MD, 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, in the press release. “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.”

Source: Spark Therapeutics