NICE recommends Luxturna to treat RPE65-mediated inherited retinal dystrophies.- Novartis

The National Institute for Health and Care Excellence (NICE) has recommended the use of Luxturna (voretigene neparvovec) . The treatment has been approved as an option for treating RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations, who have sufficient viable retinal cells. The drug is recommended based on data from a Phase I clinical trial which found that the difference in mean change in binocular MLMT score between patients treated and the control group was 1.6.

Luxturna was developed by Spark Therapeutics and is a one-time therapy indicated to treat inherited retinal dystrophies in both children and adults with vision loss caused by genetic mutations in the two copies of the RPE65 gene. The drug delivers the functional copy of the RPE65 gene to improve vision and block progressive sight loss. Its administration is via injection under the retina.