Opfolda

miglustat · Amicus Therapeutics Europe Limited
AUTHORISED IN THE EU

What Opfolda is used for

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).

Approved use, as published by the European Medicines Agency.

Key facts

Brand
Opfolda
Active substance
miglustat
Disease area
Glycogen Storage Disease Type II
Medicine group
Other alimentary tract and metabolism products
ATC code
A16AX06
Marketing authorisation holder
Amicus Therapeutics Europe Limited
Authorised in the EU since
2023

Official information

Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.

Common questions

What is Opfolda used for?

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).

What is the active substance in Opfolda?

Opfolda contains miglustat.

Who makes Opfolda?

The EU marketing authorisation holder is Amicus Therapeutics Europe Limited.

Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.

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For information only, not medical advice. This page is a plain-language summary written from standard prescribing information and the public guides linked below. Brands sometimes change their formula, so always check the composition printed on the pack. Doses here are typical adult doses; yours may differ. Do not start, stop or change a medicine without your doctor or pharmacist, and keep all medicines away from children. In an emergency, call 112 (India) or your local emergency number.