Elosulfase alfa (trade name Vimizim) is a drug for the treatment of

Morquio syndrome Morquio syndrome, also known as Mucopolysaccharidosis Type IV (MPS IV), is a rare metabolic disorder in which the body cannot process certain types of sugar molecules called glycosaminoglycans (AKA GAGs, or mucopolysaccharides). In Morquio syndr ...

which is caused by a deficiency in the enzyme

N-acetylgalactosamine-6-sulfatase The enzyme ''N''-acetylgalactosamine-6-sulfatase (EC 3.1.6.4) catalyzes the chemical reaction of cleaving off the 6-sulfate groups of the ''N''-acetyl-D-galactosamine 6-sulfate units of the macromolecule chondroitin sulfate and, similarly, of the ...

. Elosulfase alfa is a synthetic version of this enzyme. Elosulfase alfa was developed by

BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme re ...

and approved for use in the US by the

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

in 2014. The drug is used in

enzyme replacement therapy Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is availab ...

; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A. Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper. 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. In June 2019, a Belgian court issued a

preliminary injunction An injunction is a legal and equitable remedy in the form of a special court order that compels a party to do or refrain from specific acts. ("The court of appeals ... has exclusive jurisdiction to enjoin, set aside, suspend (in whole or in par ...

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 Reimbursement is the act of compensating someone for an out-of-pocket expense by giving them an amount of money equal to what was spent. Companies, governments and nonprofit organizations may compensate their employees or officers for necessary a ...

of the product repeatedly failed. This caused the parents to start

legal proceeding Legal proceeding is an activity that seeks to invoke the power of a tribunal in order to enforce a law. Although the term may be defined more broadly or more narrowly as circumstances require, it has been noted that " e term ''legal proceedings'' i ...

s 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.

References

Peptides {{gastrointestinal-drug-stub