NICE recommends gene therapy for rare eye disease

Novartis’ Luxturna has been recommended as an option for treating patients with RPE65-mediated retinal dystrophies

Patients with a rare genetic eye condition could receive gene therapy funded by the NHS after the National Institute for Health and Care Excellence recommended Luxturna.

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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