Avalglucosidase alfa for treating Pompe disease

2.1 Avalglucosidase alfa (AVAL; Nexviadyme, Sanofi Genzyme) is indicated 'for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid alpha-glucosidase deficiency).'

2.2 The dosage schedule is available in the summary of product characteristics for AVAL.

2.3 The list price of a 100‑mg vial of AVAL is £783.33 (excluding VAT; company submission).

2.4 The company has a commercial arrangement. This makes AVAL available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.