Basic Information
Mepsevii
Regulatory Information
EMEA/H/C/004438
August 23, 2018
April 26, 2018
8
June 28, 2024
Company Information
Germany
Rahel-Hirsch-Str. 10 10557 Berlin
Ultragenyx Germany GmbH
Drug Classification
Active Substances Detail
EMA Resources
Detailed Information
Therapeutic Indication
### Therapeutic indication Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
Overview Summary
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.