NICE recommends novel gene therapy

New NICE guidance has recommended voretigene neparvovec (Luxturna) for people with inherited RPE65-mediated retinal dystrophies.

RPE65-mediated inherited retinal dystrophies are rare and serious. They involve progressive loss of vision which ultimately leads to near-total blindness. Injected directly into the retina by a surgeon, the therapy introduces a healthy copy of the gene into the retinal cells, enabling the production of functional RPE65 protein.

Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said: ‘This is a highly innovative treatment for a very distressing, inherited condition so we are pleased [Novartis Pharmaceuticals UK] has worked with us, NHS England and NHS Improvement, to allow us to offer families hope for the future.’