1.1 Voretigene neparvovec is recommended, within its marketing authorisation, 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 and who have sufficient viable retinal cells. It is recommended only if the company provides voretigene neparvovec according to the commercial arrangement.
Why the committee made these recommendations
RPE65-mediated inherited retinal dystrophies are rare and serious. They involve progressive loss of vision. This ultimately leads to near-total blindness, and severely affects the quality of life of people with the condition, and their families and carers. Current treatment is supportive care.
Clinical trial evidence shows that, in the short term, voretigene neparvovec improves vision and prevents the condition from getting worse. There is no long-term clinical evidence, but it is biologically plausible that the treatment effect is likely to continue for decades.
Some assumptions in the economic modelling are uncertain, particularly around the utility values and how long the treatment effect lasts. Despite the uncertainties, voretigene neparvovec is likely to provide important clinical benefits for people with RPE65-mediated inherited retinal dystrophies, and is considered an appropriate use of NHS resources within the context of a highly specialised service. It is therefore recommended for use in the NHS.