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NICE recommends Avalglucosidase alfa as a treatment option for Pompe.

24th August 2022

The National Institute for Health and Care Excellence today published its recommendation of Avalglucosidase alfa (AVAL) as an option for treating Pompe in babies, children, young people and adults, where AVAL is provided according to the commercial arrangement with the company.

The recommendation is the outcome a NICE technology appraisal process which carefully reviewed the clinical evidence available to date and submissions from key stakeholders.

Along with evidence submitted by Sanofi Genzyme and by clinical experts, the appraisal committee considered an AGSD-UK submission setting out survey responses from the Pompe community about the impact of the condition. The committee also heard from our nominated patient experts about the effect Pompe has on their lives and experience of using AVAL.

In their published summary of the benefits AVAL is expected to provide, NICE stated:

“Avalglucosidase alfa (AVAL) is indicated for the long-term treatment of Pompe disease. AVAL is expected to provide benefits as a treatment option for IOPD and LOPD. Clinical experts explained that AVAL is the same enzyme as ALGLU but has a better delivery mechanism which should get more enzyme into muscle cells. Therefore, they expect AVAL to have a positive effect for people with Pompe disease and be a better option than ALGLU. People with Pompe disease are optimistic about future treatment with AVAL. One person who has had treatment with AVAL told of the positive effect it has had on their life. Since treatment with AVAL in the clinical trial, they no longer have mobility or breathing problems, and do not have to worry about not being able to do things that people without the disease may be able to do. The committee concluded that clinicians and people with Pompe disease would welcome an effective alternative to current treatment.”


Further details of the NICE recommendation can be found here:

NICE website