Pombiliti
What Pombiliti is used for
Pombiliti (cipaglucosidase alfa) is 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 alpha-glucosidase [GAA] deficiency).
Approved use, as published by the European Medicines Agency.
Key facts
- Brand
- Pombiliti
- Active substance
- cipaglucosidase alfa
- Disease area
- Glycogen Storage Disease Type II
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AB
- Marketing authorisation holder
- Amicus Therapeutics Europe Limited
- Authorised in the EU since
- 2023
Official information
In the USA
Same active substance: Pombiliti Atga
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 Pombiliti used for?
Pombiliti (cipaglucosidase alfa) is 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 alpha-glucosidase [GAA] deficiency).
What is the active substance in Pombiliti?
Pombiliti contains cipaglucosidase alfa.
Who makes Pombiliti?
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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