Gene therapy for rare eye disease set to be offered on NHS

Posted: Wednesday 04 September 2019
Close up of young boys eye

A gene therapy for a rare inherited eye disorder which can improve vision could soon be available on the NHS.

Today the National Institute for Health and Care Excellence (NICE) has recommended Luxturna (voretigene neparvovec) for use in the NHS in England, making it the first available treatment for an inherited retinal dystrophy.

People with a mutation of the RPE65 gene will be suitable for the new treatment. The gene should provide the instructions to make a protein that is key to normal vision.

In the gene therapy, a healthy copy of the gene is injected directly into the eye so a working protein can be produced. Patients have to have some functioning retinal cells for it to work.

Research has shown that, in the short term that the drug improves vision and prevents the condition from getting worse.

The draft recommendations are now out for consideration by the company, healthcare professionals and patient groups. If there are no appeals, NICE expects to publish its final guidance next month. The treatment would then become available from January 2020.

The list price is high, at £613,410 per patient, but the NHS has done a deal with Novartis, the UK supplier of the drug, and it is estimated that just under 90 people in England will be eligible for the treatment.

What does this mean for people with macular disease?

This treatment is only for the treatment of Leber congenital amaurosis type 2 (LCA2) and severe early-onset RP caused by mutations in a specific gene called RPE65.

However, it is a fantastic breakthrough as Luxturna becomes the first gene therapy for eye disease.

Researchers are hopeful this could pave the way for similar treatments for dry age-related macular degeneration (AMD) to be made available on the NHS.

While there is currently no such treatment for macular disease a therapy is being trialled by Professor Robert MacLaren, Professor of Ophthalmology at the University of Oxford.

The clinical trial treated its first patient earlier this year.

The trial is sponsored by Gyroscope Therapeutics