Elosulfase alfa explained

Tradename:	Vimizim
Licence Eu:	yes
Dailymedid:	Elosulfase alfa
Routes Of Administration:	Intravenous
Atc Prefix:	A16
Atc Suffix:	AB12
Legal Au:	S4
Legal Au Comment:	^[1]
Legal Us:	Rx-only
Cas Number:	9025-60-9
Unii:	ODJ69JZG85
Iuphar Ligand:	7392
Chemspiderid:	none
Kegg:	D10333
C:	5020
H:	7588
N:	1364
O:	1418
S:	34

Elosulfase alfa, sold under the brand name Vimizim, is a medication used 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]

Elosulfase alfa 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]

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.^[5]

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.^[6]

Notes and References

1. Web site: Prescription medicines: registration of new chemical entities in Australia, 2014 . Therapeutic Goods Administration (TGA) . 21 June 2022 . 10 April 2023.
2. Web site: FDA approves Vimizim to treat rare congenital enzyme disorder . . Feb 14, 2014 . December 16, 2019 . January 27, 2017 . https://web.archive.org/web/20170127011259/http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm386008.htm . dead .
3. Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, Lin SP, Mengel E, Scarpa M, Valayannopoulos V, Giugliani R, Slasor P, Lounsbury D, Dummer W . 6 . 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 . November 2014 . 24810369 . 4206772 . 10.1007/s10545-014-9715-6 .
4. Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, Parini R, Hughes D, Pastores GM, Lau HA, Al-Sayed MD, Raiman J, Yang K, Mealiffe M, Haller C . 6 . Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial . Molecular Genetics and Metabolism . 114 . 2 . 178–85 . February 2015 . 25284089 . 10.1016/j.ymgme.2014.08.012 . free .
5. Web site: 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 . 13 September 2018 . 14 April 2018 . https://web.archive.org/web/20180414212306/http://www.health.gov.au/internet/ministers/publishing.nsf/Content/health-mediarel-yr2017-hunt061.htm . live .
6. Web site: BioMarin moet verder levensreddend middel leveren aan patiënte. 26 June 2019. www.tijd.be. De Tijd. Dutch. BioMarin must continue to provide life-saving drug to patient. 26 June 2019. 27 June 2019. https://web.archive.org/web/20190627100653/https://www.tijd.be/ondernemen/farma-biotech/biomarin-moet-verder-levensreddend-middel-leveren-aan-patiente/10140276.html. live.