Ad Comm Gives Thumbs Up to Gene Therapy for Inherited Eye Disease

An FDA advisory committee unanimously endorsed Spark Therapeutics’ gene therapy for vision loss.

The 16-0 vote by the Cellular, Tissue, and Gene Therapies Advisory Committee came in response to the question of whether Spark’s Luxturna  (voretigene neparvovec) has “an overall favorable risk-benefit profile” for treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, an inherited condition that can lead to progressive deterioration of vision and total blindness.

The vote came after the committee heard testimony on the life-changing effects of the medication from several patients who had received it. The committee also reviewed evidence from a Phase 3 clinical trial.