NICE recommends gene therapy for rare eye disease
Novartis’ Luxturna has been recommended as an option for treating patients with RPE65-mediated retinal dystrophies
The Novartis treatment will be available on the NHS for patients with RPE65-mediated retinal dystrophies.
The therapy works by replacing the mutated RPE-65 gene with a working copy. It is administered as a single injection below the retina in patients who have confirmed RPE-65 mutations and viable retinal cells.
Country president of Novartis UK, Haseeb Ahmad, highlighted: “Through effective collaboration with NICE and NHS England, it has been possible to secure rapid access to the first and only one-time gene therapy for patients living with this condition.”
Luxturna is the first EU-licensed treatment for RPE-65 mutations.
Speaking at the Health Innovation Expo conference in Manchester (4–5 September), NHS chief executive Simon Stevens said: “Loss of vision can have devastating effects, particularly for children and young people, but this is a truly life changing treatment that restores the sight of people with this rare and distressing condition.”
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