The National Institute for Health and Care Excellence (NICE) has recommended the use of Luxturna (voretigene neparvovec) on the NHS, Novartis has announced.
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 injected under the retina and carries a functioning RPE65 gene to act in place of the faulty one, and 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.
The recommendation means that it will be the only treatment available in England and Wales for adults and children with the rare progressive genetic condition, which is characterised by loss of light sensitivity, loss of peripheral vision to loss of sharpness or clarity of vision and potential progression to total blindness.
Novartis announced that it is “delighted” with the decision, as Tina Houlihan, chief executive at Retina UK commented “The patient burden is high for those born with a mutation in both copies of the RPE65 gene. The progressive and debilitating nature of this rare genetic condition places a life-long physical, emotional and financial burden on patients and their families.
“NICE’s recommendation of Luxturna marks a pivotal moment as, for the first time, children and adults born with this condition have a much needed treatment option.”
Currently in the UK, it is estimated that 180 patients have mutations in both copies of the RPE65 gene, with less than 50% diagnosed following a genetic test.