European Commission approves Luxturna to treat patients with inherited retinal disease

23 November 18

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Press Office

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The European Commission has granted Voretigene neparvovec (Luxturna) market authorisation for the treatment of patients within the EU with an inherited retinal dystrophy (IRD) caused by RPE65 gene mutation.

IRDs are a group of eye diseases caused by mutations in different genes. Over 200 different genes have been identified which lead to sight loss. There are currently no treatments available to patients for any of these conditions on the NHS.

This is the first licensed gene therapy to be granted market authorisation by the European Medicines Agency (EMA) for any eye condition. NICE and the other UK regulatory bodies will have to appraise Luxturna before it can be made available for patients on the NHS.

Dr Neil Ebenezer, Director of Research, Policy and Innovation at Fight for Sight, said: “We welcome the approval of Luxturna by the European Commission as it signals a significant step change in the treatment of genetic eye conditions that are currently untreatable. We will engage with the process that regulates access to treatments for patients in the UK for this novel therapy.”

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