cipaglucosidase alfa

This medicine is authorised for use in the European Union.


Pombiliti is a medicine used to treat adults with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency), an inherited disorder in which patients have breathing difficulties and muscle weakness. Pombiliti is used in combination with another medicine, miglustat.

Pombiliti contains the active substance cipaglucosidase alfa.

This EPAR was last updated on 16/05/2023

Authorisation details

Product details
Agency product number
Active substance
Cipaglucosidase alfa
International non-proprietary name (INN) or common name
cipaglucosidase alfa
Therapeutic area (MeSH)
Glycogen Storage Disease Type II
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Amicus Therapeutics Europe Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Amicus Therapeutics Europe Limited
Block 1, Blanchardstown Corporate Park
Ballycoolin Road
Blanchardstown, Dublin
D15 AKK1

Product information

Pombiliti - EMEA/H/C/005703 -

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

Pharmacotherapeutic group

Other alimentary tract and metabolism products

Therapeutic indication

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 α-glucosidase [GAA] deficiency).

Assessment history

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