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NICE recommends Novartis' gene therapy Luxturna for inherited retinal dystrophy

Published on 04/09/19 at 11:26am

NICE has chosen to recommend the use of Novartis’ one-time gene therapy Luxturna (voretigene neparvovec) for use on the NHS in England and Wales for the treatment of RPE65-mediated inherited retinal dystrophies, marking the first therapy of its kind available in these regions. 

The drug watchdog’s ruling concerns patients with vision loss as a result of inherited retinal dystrophy from confirmed biallelic RPE65 mutations, and who have sufficient viable retinal cells. These patients can be affected by a range of symptoms including loss of peripheral vision, light sensitivity and clarity of vision, as well as night blindness, with a risk of eventually developing total blindness. It is estimated that this accounts for around 180 patients in the UK, with less than half of that number having reached diagnosis through a genetic test.

Through close collaboration between Novartis and NICE, the time taken to reach a decision on access to the therapy was reduced from an average of 38 weeks to just 20 weeks. It was based on Phase 1 trial data and a follow-up study, as well as Phase 3 data showing a clinically meaningful change from baseline in binocular multi-luminance mobility test score over one year of treatment.

“The progression of inherited retinal degeneration caused by RPE65 gene mutations leads to blindness, which has a profound effect on the lives of affected patients and their carers” said Robert MacLaren, Professor of Ophthalmology at the University of Oxford and Consultant Ophthalmologist at the Oxford Eye Hospital. “Until now, patients had no other pharmacological treatment options and I am absolutely delighted with the decision by NICE to recommend this one-time gene therapy. As a clinician, I believe the true value of voretigene neparvovec is its potential to improve vision in children and adults, and enabling them to participate fully at school, work and in their private lives.”

Haseeb Ahmad, Country President of Novartis UK and Managing Director (UK, Ireland and Nordics) of Novartis Pharmaceuticals, added: “We are delighted with today’s decision by NICE to recommend voretigene neparvovec for use in patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. 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.”

Matt Fellows

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