NICE draft backs Novartis eye disease gene therapy Luxturna

A novel  gene therapy treatment for a rare inherited eye disorder has been recommended by the UK’s National Institute for Health and Care Excellence (NICE) for use on the National Health Service (NHS).

The draft highly specialized technologies guidance recommends that voretigene neparvovec, trade named Luxturna and made by Novartis (NOVN: VX) for people with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations and who have enough viable retinal cells. Luxturna was approved in Europe last November.

The list price of voretigene neparvovec is £613,410 ($747,747) per patient, which is less than the $850,000 per-patient US list price. This technology breached the Budget Impact Test, meaning it would have cost the NHS more than £20 million in one of the first three years after being introduced. However, the company has a commercial arrangement with NHS England making the treatment available to the NHS at a discount, the size of which is commercial in confidence.