First UK patients get Novartis' Luxturna gene therapy for blindness
The first NHS patients have started treatment with Novartis Luxturna, a gene therapy for a sight-robbing inherited disease, after the drugmaker agreed a discount on its £613,000 list price last year.
Luxturna (voretigene neparvovec) was recommended for NHS funding in September for use in patients with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations, including children with Lebers congenital amaurosis (LCA).
At the time, cost-effectiveness watchdog NICE said around 86 people in England would be eligible for the treatment, which has been shown in trials to prevent progressive sight loss and even improve vision in some patients. NHS England now says that number could be as high as 100.
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The therapy is however only suitable for patients who retain some degree of vision, and there needs to be sufficient retinal cells remaining to take up and express the gene therapy.
https://pharmaphorum.com/news/first-uk-patients-get-novartis-luxturna-gene-therapy-for-blindness