Avalglucosidase alfa Fast Track - access to treatments

Avalglucosidase alfa is a potential treatment for Pompe disease developed by Sanofi Genzyme.

MDUK took part in a NICE Scoping Workshop in September 2020, ahead of which we liaised with other patient groups and with clinicians.

 We are awaiting next steps and aim to work in partnership with the Association of Glycogen Storage Diseases to submit the patient voice as part of any future consultation/appraisal.

 More information on NICE’s assessment of avalglucosidase alfa can be found at https://www.nice.org.uk/