Chemical Component Summary

Name(2R,3R,4R,5S)-1-BUTYL-2-(HYDROXYMETHYL)PIPERIDINE-3,4,5-TRIOL
Identifiers(1R,2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
FormulaC10 H21 N O4
Molecular Weight219.278
TypeNON-POLYMER
Isomeric SMILESCCCC[N@@]1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O
InChIInChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1
InChIKeyUQRORFVVSGFNRO-UTINFBMNSA-N

Chemical Details

Formal Charge0
Atom Count36
Chiral Atom Count4
Bond Count36
Aromatic Bond Count0

Drug Info: DrugBank

DrugBank IDDB00419 
NameMiglustat
Groups approved
DescriptionMiglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, 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. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect.
Synonyms
  • Miglustatum
  • BuDNJ
  • Miglustat hydrochloride
  • 1,5-(butylimino)-1,5-dideoxy, D-glucitol
  • AT2221
Brand Names
  • Opfolda
  • Miglustat Dipharma
  • Sandoz Miglustat
  • Zavesca
  • Miglustat Gen.orph
IndicationFor the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C).
Categories
  • Alimentary Tract and Metabolism
  • Alkaloids
  • Anti-HIV Agents
  • Anti-Infective Agents
  • Carbohydrates
ATC-CodeA16AX06
CAS number72599-27-0

Drug Targets

NameTarget SequencePharmacological ActionActions
Ceramide glucosyltransferaseMALLDLALEGMAVFGFVLFLVLWLMHFMAIIYTRLHLNKKATDKQPYSKL...unknowninhibitor
Drug Info/Drug Targets: DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Knox C, Law V, Jewison T, Liu P, Ly S, Frolkis A, Pon A, Banco K, Mak C, Neveu V, Djoumbou Y, Eisner R, Guo AC, Wishart DS. Nucleic Acids Res. 2011 Jan; 39 (Database issue):D1035-41. | PMID:21059682

Related Resource References

Resource NameReference
Pharos CHEMBL1029
PubChem 51634
ChEMBL CHEMBL1029
ChEBI CHEBI:50381