Awaiting funding decision for Luxturna

Posted on: Wednesday 14 August 2019

In the last issue of Look Forward, we reported that the gene therapy Luxturna had won European regulatory approval for the treatment of a
particular type of inherited sight loss.

Luxturna is for the treatment of Leber Congenital Amaurosis type 2 (LCA2) and severe early-onset RP caused by mutations in a gene called RPE65. People must have two faulty copies of RPE65, confirmed by genetic testing, as well as reasonable numbers of remaining viable retinal cells, in order to benefit from this treatment. Due to the severe, early-onset nature of this type of inherited retinal disease, eligible patients are likely to be children.

The availability of Luxturna through the NHS has yet to be decided on, so nobody can currently access it in the UK.

Luxturna is currently undergoing appraisal by the National Institute for Health & Care Excellence (NICE) and the Scottish Medicines Consortium (SMC), which provide guidance to NHS England and NHS Scotland about which treatments they should fund. (Wales and Northern Ireland are likely to follow England’s lead.)

Retina UK is actively involved in this process; we are working hard to make sure that our community’s voice is heard and that NICE and the SMC fully understand the enormous difference that an effective gene therapy could make to the lives of young people with inherited sight loss.

We expect NICE to publish their formal guidance on NHS funding in December 2019. If the outcome is positive, the NHS will need to establish a pathway for treatment and commission a small number of specialist treatment centres. All being well, Luxturna may be
available on the NHS from spring 2020.

We don’t yet know if Luxturna will be accessible through private healthcare, although we will keep you updated with any information as soon as we have it. Administration of Luxturna involves injection into the retina, requiring highly specialised facilities; this might present a challenge outside of NHS settings. Given the high cost of development and manufacture of gene therapies, the cost is likely to be in the region of several hundred thousand pounds.