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.
Therapeutic Indication
### Therapeutic indication Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome).
Therapeutic Area (MeSH)
N/AATC Code
A16AB18
ATC Item
vestronidase alfa
Pharmacotherapeutic Group
Enzymes
Active Substance (Summary)
INN / Common Names
N/A| Substance | CAS | Monograph |
|---|---|---|
| vestronidase alfa | N/A | Vestronidase alfa |
EMA Name
Mepsevii
Medicine Name
Mepsevii
Aliases
N/ANo risk management plan link.
