Naglazyme (galsulfase)
- Region
- 🇪🇺 EU
- Active substance
- galsulfase
- Company
- BioMarin International Limited
- Status
- Authorised
- Therapeutic area
- Mucopolysaccharidosis VI
- EU approval
- Mon 23 Jan 2006
- Application
- EMEA/H/C/000640
- ATC
- A16AB
- Official record
- www.ema.europa.eu
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.
Regulatory milestones
No milestones recorded in the last year.
Follow Naglazyme
Get VigiEye Daily: approvals, opinions, safety actions and PDUFA dates every morning.
Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.