Novartis has announced that the Committee for Medicinal Products for Human Use (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.
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’re 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 3 trial that together enrolled 43 patients with inherited retinal disease caused by mutations in both copies of the RPE65 gene.
Christina Fasser, president of Retina International, an umbrella organisation of more than 43 patient organisations worldwide promoting research to find treatments for inherited retinal degenerative diseases said, “Inherited retinal diseases are a heterogenous group of degenerative conditions that represent the major cause of blindness in childhood and active working life. This opinion represents a hopeful milestone for current and future patients who may ultimately benefit from gene therapy.”
Paul Hudson, CEO, Novartis Pharmaceuticals said, “Today’s positive CHMP opinion represents a significant step in our journey toward advancing potentially life-changing cell and gene therapies in ophthalmology. We look forward to working with Spark Therapeutics and the EMA to establish access and reimagine care for people in the EU who face the threat of total blindness from this inherited retinal disease.”