This is a summary of the European public assessment report (EPAR) for Naglazyme. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Naglazyme.
Therapeutic Indication
Naglazyme is indicated for long\-term enzyme\-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N\-acetylgalactosamine\-4\-sulfatase deficiency; Maroteaux\-Lamy syndrome) (see section 5\.1\). As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non\-reversible clinical manifestations of the disease. A key issue is to treat young patients aged \<5 years suffering from a severe form of the disease, even though patients \<5 years were not included in the pivotal phase\-3 study.
Therapeutic Area (MeSH)
ATC Code
A16AB
ATC Item
N/A
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| galsulfase | N/A | galsulfase |
EMA Name
Naglazyme
Medicine Name
Naglazyme
Aliases
N/ANo risk management plan link.
