Elosulfase alfa

Elosulfase alfa
Clinical data
Trade names Vimizim
AHFS/Drugs.com Multum Consumer Information
Pregnancy
category
  • US: C (Risk not ruled out)
Legal status
Legal status
Identifiers
CAS Number 9025-60-9
ATC code A16AB12 (WHO)
IUPHAR/BPS 7392
ChemSpider none
Chemical data
Formula C5020H7588N1364O1418S34
Molar mass 110.8 kg/mol

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]

References


This article is issued from Wikipedia - version of the Tuesday, April 05, 2016. The text is available under the Creative Commons Attribution/Share Alike but additional terms may apply for the media files.