Novartis’ gene therapy Luxturna for a rare, inherited form of blindness should be available for routine NHS use in England and Wales, says NICE.
The one-off treatment – which was approved in Europe last November – has been backed by the cost-effectiveness watchdog for use in children and adults who are living with vision loss due to genetic mutations in both copies of the RPE65 gene.
These patients almost invariably end up losing their sight entirely and have no approved therapies to hold up the decline. Luxturna (voretigene neparvovec) has been shown in clinical trials to both improve vision and prevent progressive sight loss.
Mutations in both copies of the RPE65 gene affect approximately 1 in 200,000 people, and it is estimated there are around 180 patients with double mutations in the RPE65 gene in England and Wales, with fewer than half diagnosed via a genetic test.
Patients also have to have sufficient retinal cells remaining for the treatment – which involves a one-time injection under the retina – to be carried out. According to the draft guidance, the company and NHS England have agreed a confidential discount on the list price of £613,410 for the therapy.
Sue Sharp, deputy chief executive at the Royal Society for Blind Children, said: “We see first-hand the devastating effect of childhood sight loss, and so we welcome news of this breakthrough therapy and its impact on the lives of children with inherited retinal dystrophies disorder.”
The Scottish Medicines Consortium (SMC) is due to deliver its verdict on the gene therapy in December.
“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,” said Haseeb Ahmad, managing director for UK, Ireland and Nordics at Novartis Pharma.
“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.”
Novartis secured rights to the therapy outside the US from its original developer Spark Therapeutics last year shortly after the biotech won FDA approval for Luxturna as the first directly-administered gene therapy in the US.
Since then Roche has been attempting to take over Spark entirely in a deal valued at $4.3bn, but has been forced to announce multiple delays – including another this week – that have set the completion deadline back to 30 April 2020. Antitrust concerns by regulators in the US and UK seem to be behind the hold-up.