Miglustat

From Wikipedia, the free encyclopedia

Miglustat
Systematic (IUPAC) name
1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Identifiers
CAS number 72599-27-0
ATC code A16AX06
PubChem 51634
DrugBank APRD01118
Chemical data
Formula C10H21NO4 
Mol. mass 219.28 g/mol
Pharmacokinetic data
Bioavailability 97%
Protein binding Nil
Metabolism Nil
Half life 6–7 hours
Excretion Renal, unchanged
Therapeutic considerations
Licence data

US
Pregnancy cat.

X(US)
Legal status

℞-only(US)
Routes Oral

Miglustat is a drug used to treat Gaucher disease.

It inhibits the enzyme glucosylceramide synthase,[1] an essential enzyme for the synthesis of most glycosphingolipids.

It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. [2]

It is marketed under the trade name Zavesca.

It has also been investigated for use in treating Niemann-Pick disease.[3]

[edit] References

  1. ^ van Giersbergen PL, Dingemanse J (2007). "Influence of Food Intake on the Pharmacokinetics of Miglustat, an Inhibitor of Glucosylceramide Synthase". doi:10.1177/0091270007305298. PMID 17720777. 
  2. ^ Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P (2005). "Guidance on the use of miglustat for treating patients with type 1 Gaucher disease". Am. J. Hematol. 80 (3): 223–9. doi:10.1002/ajh.20504. PMID 16247743. 
  3. ^ Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE (2007). "Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study". Lancet neurology 6 (9): 765–72. doi:10.1016/S1474-4422(07)70194-1. PMID 17689147. 
Tablets  This pharmacology-related article is a stub. You can help Wikipedia by expanding it.
v • d • e
Other alimentary tract and metabolism products (A16)
Amino acids and derivatives
Enzymes
Various alimentary tract
and metabolism products