Pombiliti
cipaglucosidase alfa
Table of contents
Overview
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.
-
List item
Pombiliti : EPAR - Medicine Overview (PDF/148.11 KB) (new)
First published: 16/05/2023
EMA/950090/2022 -
-
List item
Pombiliti : EPAR - Risk management plan summary (PDF/394.94 KB) (new)
First published: 16/05/2023
Authorisation details
Product details | |
---|---|
Name |
Pombiliti
|
Agency product number |
EMEA/H/C/005703
|
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 |
A16AB
|
Additional 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. |
Orphan |
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 |
20/03/2023
|
Contact address |
Amicus Therapeutics Europe Limited |
Product information
Pombiliti - EMEA/H/C/005703 -
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
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).