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Mepsevii is a medicine to treat mucopolysaccharidosis type VII (MPS VII, also known as Sly syndrome), an inherited disease caused by a lack of an enzyme needed to break down complex carbohydrates known as glycosaminoglycans (GAGs).
The disease leads to build up of GAGs in the body, which causes a wide range of problems, including joint stiffness, short stature, enlarged liver and spleen, hearing loss, cataract and delays in development.
MPS VII is rare, and Mepsevii was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2012.
Mepsevii : EPAR - Medicine overview (PDF/113.24 KB)
First published: 28/08/2018
Last updated: 13/10/2020
Mepsevii : EPAR - Risk-management-plan summary (PDF/100.61 KB)Adopted
First published: 28/08/2018
Last updated: 28/08/2018
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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 authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.
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.
Ultragenyx Germany GmbH
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21/12/2021 Mepsevii - EMEA/H/C/004438 - N/0028
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.
Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).