title: Miglustat. Oxford GlycoSciences/Actelion.
abstract: Oxford GlycoSciences and Actelion have developed and launched miglustat (OGT-918; Vevesca; Zavesca) for the treatment of type 1 Gaucher disease. Miglustat is an orally administered small-molecule glucosylceramide glucosyltransferase inhibitor licensed from Searle, and it is also in development for the treatment of other glycolipid storage disorders such as Tay-Sachs, Fabry and Niemann-Pick type C diseases. In November 2002, miglustat received EU approval for the treatment of Gaucher disease and was launched in the UK in March 2003 by Actelion. At this time, additional EU launches were expected over the next few months.
authors.affiliation: University of Cambridge, Department of Medicine, Addenbrooke's Hospital, Hills Road, Cambridge, CB2 2QQ, UK. rhl20@cam.ac.uk
authors.forename: Robin H
authors.initials: RH
authors.lastname: Lachmann
mesh_terms: D000818:Animals; D002239:Carbohydrate Metabolism, Inborn Errors / Q000188:drug therapy*; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D000795:Fabry Disease / Q000188:drug therapy; D005776:Gaucher Disease / Q000188:drug therapy; D005962:Glucosylceramidase / Q000009:adverse effects / Q000138:chemical synthesis / Q000378:metabolism / Q000494:pharmacology / Q000627:therapeutic use* / Q000633:toxicity; D006801:Humans; D009542:Niemann-Pick Diseases / Q000188:drug therapy; D013329:Structure-Activity Relationship
chemical_list: D005962:Glucosylceramidase; C090568:imiglucerase