Posted by Michael Wonder on 23 Nov 2018
Novartis announces landmark EU approval for one-time gene therapy Luxturna to restore vision in people with rare inherited retinal disease
23 November 2018 - Luxturna (voretigene neparvovec) is the first gene therapy to treat an inherited retinal disease, indicated for children and adults with vision loss caused by mutations in both copies of the RPE65 gene and sufficient viable retinal cells.
Novartis announced today that the European Commission approved 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 and who have enough viable retinal cells. The authorisation is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway. Luxturna was developed and is commercialised in the US by Spark Therapeutics.
The EC decision is based on a positive CHMP opinion that looked at data from a Phase 1 clinical trial, its follow-up trial, and the first randomised, controlled Phase 3 gene therapy trial for an inherited disease.
