Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease

2.1 Cipaglucosidase alfa (Pombiliti, Amicus Therapeutics Ltd) is indicated as 'a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency)'. Miglustat (Opfolda, Amicus Therapeutics Ltd) is indicated as 'an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency)'.

2.2 The dosage schedules are available in the summary of product characteristics for cipaglucosidase alfa and the summary of product characteristics for miglustat.

2.3 The list price of cipaglucosidase alfa is £987 per 105 mg vial (excluding VAT; company communication). The list price of miglustat is £116.69 per 4 capsules of 65 mg (excluding VAT; company submission).

2.4 The company for cipaglucosidase alfa has a commercial arrangement. This makes it 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.