Gene therapy treatment Luxturna accepted for use in Scotland

Retina UK is delighted that the Scottish Medicines Consortium (SMC) has accepted the gene therapy Luxturna (voretigene neparvovec) for ongoing use by the NHS in Scotland.

Luxturna is designed specifically for treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene. It is a one-off treatment injected into the eye under general anaesthetic and is most effective if given relatively early in the course of the condition, when it can potentially slow or even stop progression of sight loss and in some cases may lead to improvements in vision. More information about Luxturna can be found on our Luxturna Frequently Asked Questions page. The treatment will not work for inherited retinal conditions associated with any gene other than RPE65.

Luxturna was accepted for use by the NHS in England, Wales and Norther Ireland in 2019, but at that point was placed under review by the SMC as part of its special framework for very rare conditions. This meant that it was initially offered in Scotland for a limited period only, and required reassessment this year when further data on its impact were available.

Retina UK was actively involved in the recent SMC decision-making process, representing the inherited sight loss community and highlighting the urgent need for effective treatments. The successful outcome of the reassessment means that Luxturna will now be permanently available for prescription in Scotland.

Tina Garvey, Chief Executive of Retina UK, said: ““Today’s decision is an important step forward for the inherited sight loss community in Scotland. The relentless progression of vision loss takes an immense psychological toll, with over 90% of respondents to our surveys reporting anxiety, depression, stress and loneliness directly attributed to their retinal condition. We are so pleased that some members of our community now have access to a potentially life-changing therapy, but for most, there are still no options. Retina UK will continue to drive forward innovative research while supporting affected families.”

Dr Roly Megaw, Honorary Consultant Ophthalmologist for NHS Lothian and clinical lecturer at Edinburgh University, said: “Families living with inherited retinal conditions are in desperate need of treatments. I am so pleased that people in Scotland affected by RPE65-associated sight loss will have the option to receive Luxturna, a cutting-edge therapy that can significantly improve visual function.”