Elosulfase alfa
| Clinical data | |
|---|---|
| Trade names | Vimizim |
| AHFS/Drugs.com | Multum Consumer Information |
| Pregnancy category |
|
| Legal status | |
| Legal status |
|
| Identifiers | |
| CAS Number | 9025-60-9 |
| ATC code | A16AB12 (WHO) |
| IUPHAR/BPS | 7392 |
| ChemSpider | none |
| Chemical data | |
| Formula | C5020H7588N1364O1418S34 |
| Molar mass | 110.8 kg/mol |
Elosulfase alfa (trade name Vimizim) is a drug for the treatment of Morquio syndrome which is caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase. Elosulfase alfa is a synthetic version of this enzyme.
Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and approved for use in the US by the Food and Drug Administration in 2014.[1]
The drug is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. [2] Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper. [3]
References
- ↑ "FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014.
- ↑ Hendriksz, C. et. al (November 2014). "Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study". Journal of Inherited Metabolic Disease 37 (6): 979–990. doi:10.1007/s10545-014-9715-6.
- ↑ Hendriksz, C. et. al (February 2015). "Multi-domain impact of elosulfase alfa in Morquio A syndrome in the pivotal phase III trial". Molecular Genetics and Metabolism 114 (2): 178–185. doi:10.1016/j.ymgme.2014.08.012.
This article is issued from Wikipedia - version of the Tuesday, April 05, 2016. The text is available under the Creative Commons Attribution/Share Alike but additional terms may apply for the media files.