Nexviazyme

Pharmaceutical company: Sanofi Genzyme

Cells need energy to function properly. We get energy from food, and it is stored within the body as glycogen. A molecule in the body, called alpha-glucosidase breaks down glycogen to release glucose, providing energy for cells. In people with late-onset Pompe disease, alpha-glucosidase is missing. This leads to a build-up of glycogen, reducing the amount of energy cells get and causing muscle weakness.

Nexviazyme mimics alpha-glucosidase, reducing the build-up of glycogen by breaking it down to release glucose. Nexviazyme comes as a liquid and is administered slowly into the bloodstream (intravenous infusion).

Nexviazyme has been recommended as a treatment option for Pompe disease in the UK and is available on the NHS. Please consult your (or child’s) clinical team for more information.

England, Wales and Northern Ireland

Read the full National Institute for Health and Care Excellence (NICE) guidance.

Scotland

Read the full Scottish Medicines Consortium (SMC) guidance.

We are proud to have played a role in ensuring Nexviazyme is available for people with Pompe disease. Throughout the assessment processes, we worked closely with partners, including the Association of Glycogen Storage Diseases, patient groups, and clinicians to attend workshops, develop written responses and represent patients’ experiences.

Evidence from the phase 3 COMET clinical trial was used in the UK assessment of Nexviazyme. This trial compared Nexviazyme to a similar treatment for Pompe disease, called Myozyme (also known as alglucosidase alfa). 100 participants with Pompe disease, aged between 16 – 78 years old, took part in the trial. Participants were either randomly assigned to receive Nexviazyme (51 participants) or Myozyme (49 participants). The trial found Nexviazyme was safe.

After 49 weeks, participants who received Nexviazyme showed improvements in measurements of breathing and walking. However, the researchers could not confirm that the improvements seen were not due to chance. Researchers refer to this as ‘not statistically significant’.

This means that the researchers cannot say for definite that treatment with Nexviazyme is more beneficial than treatment with Myozyme. However, there is no evidence to suggest Nexviazyme is less beneficial than Myozyme, suggesting that the treatments have a similar effect.

Last updated: 18/02/2025