CHMP positive for Luxturna to treat inherited retinal diseases. Novartis

Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the approval of voretigene neparvovec (Luxturna), a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. Luxturna was developed and is marketed in the US by Spark Therapeutics. If approved, voretigene neparvovec will be commercialized by Novartis in markets outside the U.S.

Inherited retinal diseases are a group of rare conditions that can lead to total blindness, often disproportionally affecting children and young adults. 50% of people with mutations in both copies of the RPE65 gene will be legally blind by the time they are 16 years old. Voretigene neparvovec provides a working copy of the RPE65 gene to act in place of the mutated RPE65 gene. This working gene has the potential to restore vision and improve sight. Outside of the US, there are currently no approved pharmacologic treatment options for vision loss caused by RPE65 mutations.

The positive CHMP opinion is based on data from a Phase 1 clinical trial, its follow-up trial, and a Phase III trial that together enrolled 43 patients with inherited retinal disease caused by mutations in both copies of the RPE65 gene. The Phase III trial was the first randomized, controlled Phase III gene therapy trial for an inherited disease.