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CHMP recommends miglustat the enzyme stabilizer component for Pombiliti for Pompe disease

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Published:28th Apr 2023

Amicus Therapeutics announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization of miglustat, the enzyme stabilizer component of AT-GAA

A decision for miglustat from the European Commission (EC) is expected in the third quarter of 2023, after which the two-component therapy will be fully approved, and the Company will begin the country-by-country reimbursement and launch process.

Miglustat will be commercialized under the brand name Opfolda. The biologic component of the two-component therapy, Pombiliti (cipaglucosidase alfa), was approved by the European Commission in March 2023.

Condition: Pompe Disease

Type: drug

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