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.
[3] Elosulfase alfa is used in enzyme replacement therapy; a 2014 study confirmed it was effective on young patients with Morquio syndrome type A.
[4] Treatment with this medication was most effective upon respiratory symptoms, activities of daily living and growth, as confirmed in a 2015 paper.
[6] 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.