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# Panel Backs Sparks Gene Therapy for Vision Loss
- URL: https://www.fdaweb.com/panel-backs-sparks-gene-therapy-for-vision-loss/
- Published: 2017-10-12T12:00:00.000Z
- Updated: 2026-09-14T22:55:45.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5139858

FDA's Cellular, Tissue and Gene Therapies Advisory Committee has voted unanimously (16 to 0) to recommend approval for a Spark Therapeutics BLA for Luxturna (voretigene neparvovec) for treating patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy. A [briefing document](https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM579290.pdf?ref=fdaweb.com) released in advance of the meeting noted that the gene therapy met the main criteria for efficacy. In the key study, the reviewers said, patients had a significant two-point or more improvement in light levels in both eyes, but checking visual acuity showed that patients failed to demonstrate a significant improvement in that secondary endpoint.

The therapy has a priority review and a 1/12/2018 user fee review action deadline. It has received orphan drug, breakthrough therapy and rare pediatric disease designations from FDA.