Elosulfase alfa

Elosulfase alfa
Clinical data
Trade names Vimizim
AHFS/Drugs.com Multum Consumer Information
License data
Pregnancy
category
  • US: C (Risk not ruled out)
    ATC code
    Legal status
    Legal status
    Identifiers
    CAS Number
    IUPHAR/BPS
    ChemSpider
    • none
    KEGG
    Chemical and physical 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]

    The cost of elosulfase alfa in some countries is $400,000 a year, which has made it difficult for some health systems to afford it.[4]

    References

    1. ↑ "FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014.
    2. ↑ 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.
    3. ↑ 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. PMID 25284089.
    4. ↑ [http://www.health.gov.au/internet/ministers/publishing.nsf/Content/health-mediarel-yr2017-hunt061.htm Providing $400,000 life-saving drug to Australian kids with rare disease. The Australian Government will provide a life-saving treatment to Australian patients who have a rare medical condition known as Morquio A Syndrome, at no cost. The Hon Greg Hunt MP. Minister for Health. 15 June 2017


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