Elosulfase alfa
| Clinical data | |
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| Trade names | Vimizim |
| AHFS/Drugs.com | Multum Consumer Information |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| IUPHAR/BPS | |
| ChemSpider |
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| Chemical and physical data | |
| Formula | C5020H7588N1364O1418S34 |
| Molar mass | 110.8 kg/mol g·mol−1 |
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.[2]
The drug is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. [3] Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper. [4]
References
- ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 Oct 2023.
- ^ "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.
{{cite journal}}: Cite has empty unknown parameter:|1=(help); Explicit use of et al. in:|author=(help) - ^ 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.
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