Posted by Michael Wonder on 22 Sep 2018
Novartis announces positive CHMP opinion for one-time gene therapy Luxturna to treat children and adults with rare inherited retinal disease
21 September 2018 - If approved in expected timeframe, Luxturna (voretigene neparvovec) will be first and only gene therapy available in EU to treat a rare inherited retinal disease.
Novartis today announced that the CHMP adopted a positive opinion for the approval of voretigene neparvovec, 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 commercialised by Novartis in markets outside the U.S.
The positive CHMP opinion is based on data from a Phase 1 clinical trial, its follow-up trial, and a Phase 3 trial that together enrolled 43 patients with inherited retinal disease caused by mutations in both copies of the RPE65 gene. The Phase 3 trial was the first randomised, controlled Phase 3 gene therapy trial for an inherited disease.
