Trade names Vimizim Legal status US: ℞-only | ATC code A16AB12 (WHO) CAS Number 9025-60-9 | |
AHFS/Drugs.com Multum Consumer Information Pregnancy
category US: C (Risk not ruled out) | ||
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.
The drug is used in enzyme replacement therapy; 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.
References
Elosulfase alfa Wikipedia(Text) CC BY-SA