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Avalgucosidase alfa

Avalglucosidase alfa (also sold under the brand name Nexviazyme) is a treatment for Pompe disease developed by Sanofi Genzyme. 

How does it work?

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.  

Availability

Since August 2022, Nexviazyme has been recommended as an option for treating Pompe disease in babies, children, young people and adults. 

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.

NICE’s recommendation means that the treatment is also available in Wales and Northern Ireland. 

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.  

In July 2023 , Nexviazyme was also accepted for use within NHS Scotland by the Scottish Medicines Consortium. More information on this is available on the SMA website here. 

Support and information

Metabolic myopathy

Pompe disease is a metabolic myopathy. Also known as metabolic muscle conditions, these are conditions that interfere with the way muscles provide energy.

Research

We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.

Our support services

We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.

Our helpline is open Monday-Thursday 10am-2pm.