Since August, Nexviazyme has been recommended as an option for treating Pompe disease in babies, children, young people and adults.
Patients with Pompe disease have an enzyme deficiency that leads to a build up of a sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness. Normally, glycogen - the stored form of glucose - breaks down to release glucose into the bloodstream to be used as fuel for the cells. Nexviazyme is an enzyme replacement therapy that helps reduce glycogen build up.
MDUK played an important role in NICE's appraisal process, as part of our commitment to increasing access to treatments for all muscle-wasting conditions. For example, we took part in a NICE Scoping Workshop in September 2020, ahead of which we liaised with other patient groups and with clinicians. In November 2021, MDUK in partnership with the Association of Glycogen Storage Diseases submitted a written response to the NICE appraisal. MDUK, working together with patients with Pompe disease, attended NICE committee meetings to discuss the benefits of the treatment and emphasise patient experiences in living with their condition.
More information on NICE’s assessment of Nexviazyme can be found here.
We are waiting to hear if Northern Ireland will mirror NICE's approval, whereas Wales will roll out the treatment around October 2022. In Scotland, Nexviazyme still needs to go through the Scottish Medicines Consortium (SMC) for approval. We will continue to monitor access to Nexviazyme across the devolved nations and we will provide an update when further information becomes available.