Novartis Gene Therapy for Vision Loss Approved in EU

The European Commission cleared Novartis’ Luxturna (voretigene neparvovec) for treatment of vision loss due to a genetic mutation in both copies of the RPE65 gene.

The treatment delivers a normal copy of the gene to retinal cells to restore vision using a modified version of a naturally-occurring adeno-associated virus.

The European Commission based its approval on a Phase 3 clinical trial showing vision improvement as early as 30 days following treatment. Vision improved for 90 percent of trial subjects.