Elosulfase alfa

Elosulfase alfa
Clinical data
Trade names	Vimizim
AHFS/Drugs.com	Multum Consumer Information
License data	EU EMA: by INN;
ATC code	A16AB12 (WHO) ;
Legal status
Legal status	US: ℞-only ;
Identifiers
CAS Number	9025-60-9;
IUPHAR/BPS	7392;
ChemSpider	none;
UNII	ODJ69JZG85;
KEGG	D10333;
Chemical and physical data
Formula	C5020H7588N1364O1418S34
Molar mass	110826.09 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.[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 $2,080,000-$6,240,000 a year, which has made it difficult for some health systems to afford it.[4]

In June 2019, a Belgian court issued a preliminary injunction forcing BioMarin to continue supplying Vimizim to a young girl suffering from Morquio syndrome free of charge. BioMarin stopped providing the drug for free at the beginning of the year after negotiations with Belgian health authorities regarding reimbursement of the product repeatedly failed. This caused the parents to start legal proceedings to force the company to keep providing the medicine free of charge. BioMarin was ordered to keep doing so until a definitive judgment would be rendered, or until the medicine would be available on the Belgian market at a reasonable price.[5]

References

"FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014.
Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, 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): 979–90. doi:10.1007/s10545-014-9715-6. PMC 4206772. PMID 24810369.
Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, et al. (February 2015). "Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial". Molecular Genetics and Metabolism. 114 (2): 178–85. doi:10.1016/j.ymgme.2014.08.012. PMID 25284089.
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
"BioMarin moet verder levensreddend middel leveren aan patiënte" [BioMarin must continue to provide life-saving drug to patient]. www.tijd.be (in Dutch). De Tijd. 26 June 2019. Retrieved 26 June 2019.

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[1] "FDA approves Vimizim to treat rare congenital enzyme disorder". Food and Drug Administration. Feb 14, 2014.

[2] Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, 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): 979–90. doi:10.1007/s10545-014-9715-6. PMC 4206772. PMID 24810369.

[3] Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, et al. (February 2015). "Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial". Molecular Genetics and Metabolism. 114 (2): 178–85. doi:10.1016/j.ymgme.2014.08.012. PMID 25284089.

[4] 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

[5] "BioMarin moet verder levensreddend middel leveren aan patiënte" [BioMarin must continue to provide life-saving drug to patient]. www.tijd.be (in Dutch). De Tijd. 26 June 2019. Retrieved 26 June 2019.