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Record Information
Version5.0
StatusExpected but not Quantified
Creation Date2012-09-06 15:16:49 UTC
Update Date2021-09-14 15:47:34 UTC
HMDB IDHMDB0014563
Secondary Accession Numbers
  • HMDB14563
Metabolite Identification
Common NameMiglustat
DescriptionMiglustat 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. It is marketed under the trade name Zavesca. 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.
Structure
Data?1582753193
Synonyms
ValueSource
BuDNJChEBI
ButyldeoxynojirimycinChEBI
MiglustatumChEBI
N-(N-Butyl)deoxynojirimycinChEBI
N-Butyl deoxynojirimycinChEBI
N-Butyl-1-deoxynojirimycinChEBI
N-ButylmoranolineChEBI
NB-DNJChEBI
SC-48334ChEBI
ZavescaChEBI
BrazavesKegg
N-(N-Butyl)deoxy-nojirimycinHMDB
N-ButyldeoxynojirimycinHMDB
OGT 918HMDB
Chemical FormulaC10H21NO4
Average Molecular Weight219.278
Monoisotopic Molecular Weight219.147058165
IUPAC Name(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Traditional Namemiglustat
CAS Registry Number72599-27-0
SMILES
CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO
InChI Identifier
InChI=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
InChI KeyUQRORFVVSGFNRO-UTINFBMNSA-N
Chemical Taxonomy
Description Belongs to the class of organic compounds known as piperidines. Piperidines are compounds containing a piperidine ring, which is a saturated aliphatic six-member ring with one nitrogen atom and five carbon atoms.
KingdomOrganic compounds
Super ClassOrganoheterocyclic compounds
ClassPiperidines
Sub ClassNot Available
Direct ParentPiperidines
Alternative Parents
Substituents
  • Piperidine
  • 1,2-aminoalcohol
  • Secondary alcohol
  • Tertiary aliphatic amine
  • Tertiary amine
  • Polyol
  • Azacycle
  • Organic nitrogen compound
  • Hydrocarbon derivative
  • Primary alcohol
  • Organopnictogen compound
  • Organic oxygen compound
  • Organooxygen compound
  • Organonitrogen compound
  • Amine
  • Alcohol
  • Aliphatic heteromonocyclic compound
Molecular FrameworkAliphatic heteromonocyclic compounds
External Descriptors
Ontology
Disposition

Biological location

Source

Route of exposure

Physical Properties
StateSolid
Experimental Molecular Properties
PropertyValueReference
Melting Point169 - 172 °CNot Available
Boiling PointNot AvailableNot Available
Water Solubility331 g/LNot Available
LogP-0.6Not Available
Experimental Spectral PropertiesNot Available
Predicted Molecular Properties
PropertyValueSource
Water Solubility331 g/LALOGPS
logP10(-1.1) g/LALOGPS
logP10(-1.2) g/LChemAxon
logS10(0.18) g/LALOGPS
pKa (Strongest Acidic)12.9ChemAxon
pKa (Strongest Basic)8.49ChemAxon
Physiological Charge1ChemAxon
Hydrogen Acceptor Count5ChemAxon
Hydrogen Donor Count4ChemAxon
Polar Surface Area84.16 ŲChemAxon
Rotatable Bond Count4ChemAxon
Refractivity55.74 m³·mol⁻¹ChemAxon
Polarizability23.99 ųChemAxon
Number of Rings1ChemAxon
BioavailabilityYesChemAxon
Rule of FiveYesChemAxon
Ghose FilterNoChemAxon
Veber's RuleNoChemAxon
MDDR-like RuleNoChemAxon
Predicted Spectral Properties

Predicted Collision Cross Sections

PredictorAdduct TypeCCS Value (Å2)Reference
DarkChem[M+H]+152.86131661259
DarkChem[M-H]-147.4831661259

Predicted Kovats Retention Indices

Derivatized

Derivative Name / StructureSMILESKovats RI ValueColumn TypeReference
Miglustat,1TMS,#1CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO1729.1Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TMS,#2CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO1713.2Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TMS,#3CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO1725.3Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TMS,#4CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C1760.7Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#1CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO1763.1Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#2CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO1781.6Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#3CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C1803.8Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#4CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO1792.3Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#5CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C1817.7Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TMS,#6CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C1813.0Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TMS,#1CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO1845.4Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TMS,#2CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C1874.8Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TMS,#3CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C1901.7Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TMS,#4CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C1903.2Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,4TMS,#1CCCCN1C[C@H](O[Si](C)(C)C)[C@@H](O[Si](C)(C)C)[C@H](O[Si](C)(C)C)[C@H]1CO[Si](C)(C)C1948.0Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TBDMS,#1CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO1985.1Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TBDMS,#2CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO1961.9Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TBDMS,#3CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO1976.4Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,1TBDMS,#4CCCCN1C[C@H](O)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C2011.8Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#1CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO2268.2Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#2CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO2277.7Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#3CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C2305.8Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#4CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO2277.8Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#5CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C2307.0Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,2TBDMS,#6CCCCN1C[C@H](O)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C2295.3Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TBDMS,#1CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO2553.4Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TBDMS,#2CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O)[C@H]1CO[Si](C)(C)C(C)(C)C2582.1Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TBDMS,#3CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C2601.5Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,3TBDMS,#4CCCCN1C[C@H](O)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C2595.6Semi standard non polarhttps://arxiv.org/abs/1905.12712
Miglustat,4TBDMS,#1CCCCN1C[C@H](O[Si](C)(C)C(C)(C)C)[C@@H](O[Si](C)(C)C(C)(C)C)[C@H](O[Si](C)(C)C(C)(C)C)[C@H]1CO[Si](C)(C)C(C)(C)C2835.9Semi standard non polarhttps://arxiv.org/abs/1905.12712
Spectra

GC-MS Spectra

Spectrum TypeDescriptionSplash KeyDeposition DateSourceView
Predicted GC-MSPredicted GC-MS Spectrum - Miglustat GC-MS (Non-derivatized) - 70eV, Positivesplash10-0zos-3910000000-487652a19fde10ffe6832017-09-01Wishart LabView Spectrum
Predicted GC-MSPredicted GC-MS Spectrum - Miglustat GC-MS (4 TMS) - 70eV, Positivesplash10-00kf-2142900000-0c0744b80c14d6b206462017-10-06Wishart LabView Spectrum
Predicted GC-MSPredicted GC-MS Spectrum - Miglustat GC-MS (Non-derivatized) - 70eV, PositiveNot Available2021-10-12Wishart LabView Spectrum

MS/MS Spectra

Spectrum TypeDescriptionSplash KeyDeposition DateSourceView
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 10V, Positive-QTOFsplash10-0fk9-0190000000-1545727fb8c9dc1b26852016-08-03Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 20V, Positive-QTOFsplash10-0ue9-6980000000-589edd008686a4fe9d4b2016-08-03Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 40V, Positive-QTOFsplash10-0a4i-9800000000-0ef03d48da73c35c0c962016-08-03Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 10V, Negative-QTOFsplash10-014r-0690000000-52d21087091279bec38b2016-08-04Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 20V, Negative-QTOFsplash10-0fe0-3930000000-e5cf8de6350860e738522016-08-04Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 40V, Negative-QTOFsplash10-00dl-9100000000-f8691ad25d535e38c6252016-08-04Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 10V, Negative-QTOFsplash10-014i-0090000000-32e6ad4c1e36e0f2fb662021-09-24Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 20V, Negative-QTOFsplash10-014i-0290000000-b7fd37a3d8a9c58a1be02021-09-24Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 40V, Negative-QTOFsplash10-0a4l-9310000000-fb18a3f257a799c2f63d2021-09-24Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 10V, Positive-QTOFsplash10-00di-0090000000-66c377bd2995bc8184382021-09-24Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 20V, Positive-QTOFsplash10-00di-2590000000-6286d923af7492795fcf2021-09-24Wishart LabView Spectrum
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - Miglustat 40V, Positive-QTOFsplash10-05gi-9000000000-ba860e30faee784f9ef12021-09-24Wishart LabView Spectrum

NMR Spectra

Spectrum TypeDescriptionDeposition DateSourceView
Predicted 1D NMR13C NMR Spectrum (1D, 100 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 100 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 1000 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 1000 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 200 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 200 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 300 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 300 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 400 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 400 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 500 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 500 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 600 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 600 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 700 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 700 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 800 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 800 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR13C NMR Spectrum (1D, 900 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Predicted 1D NMR1H NMR Spectrum (1D, 900 MHz, D2O, predicted)2021-09-25Wishart LabView Spectrum
Biological Properties
Cellular Locations
  • Membrane
Biospecimen Locations
  • Blood
  • Urine
Tissue LocationsNot Available
Pathways
Normal Concentrations
BiospecimenStatusValueAgeSexConditionReferenceDetails
BloodExpected but not QuantifiedNot QuantifiedNot AvailableNot AvailableTaking drug identified by DrugBank entry DB00419 details
UrineExpected but not QuantifiedNot QuantifiedNot AvailableNot AvailableTaking drug identified by DrugBank entry DB00419 details
Abnormal Concentrations
Not Available
Associated Disorders and Diseases
Disease ReferencesNone
Associated OMIM IDsNone
DrugBank IDDB00419
Phenol Explorer Compound IDNot Available
FooDB IDNot Available
KNApSAcK IDNot Available
Chemspider ID46764
KEGG Compound IDNot Available
BioCyc IDNot Available
BiGG IDNot Available
Wikipedia LinkMiglustat
METLIN IDNot Available
PubChem Compound51634
PDB IDNot Available
ChEBI ID50381
Food Biomarker OntologyNot Available
VMH IDNot Available
MarkerDB IDNot Available
Good Scents IDNot Available
References
Synthesis ReferenceNot Available
Material Safety Data Sheet (MSDS)Not Available
General References
  1. Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60. [PubMed:12803929 ]
  2. Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. [PubMed:16247743 ]
  3. Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18. [PubMed:19956552 ]
  4. McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. [PubMed:14609352 ]
  5. Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE: Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007 Sep;6(9):765-72. [PubMed:17689147 ]
  6. van Giersbergen PL, Dingemanse J: Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol. 2007 Oct;47(10):1277-82. Epub 2007 Aug 24. [PubMed:17720777 ]

Enzymes

General function:
Cell wall/membrane/envelope biogenesis
Specific function:
Catalyzes the first glycosylation step in glycosphingolipid biosynthesis, the transfer of glucose to ceramide. May also serve as a "flippase".
Gene Name:
UGCG
Uniprot ID:
Q16739
Molecular weight:
44853.255
References
  1. Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60. [PubMed:12803929 ]
  2. Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. [PubMed:16247743 ]
  3. Chien YH, Lee NC, Tsai LK, Huang AC, Peng SF, Chen SJ, Hwu WL: Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year. J Inherit Metab Dis. 2007 Oct;30(5):826. Epub 2007 Jun 21. [PubMed:17603755 ]
  4. Treiber A, Morand O, Clozel M: The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica. 2007 Mar;37(3):298-314. [PubMed:17624027 ]
  5. Ficicioglu C: Review of miglustat for clinical management in Gaucher disease type 1. Ther Clin Risk Manag. 2008 Apr;4(2):425-31. [PubMed:18728838 ]
  6. Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18. [PubMed:19956552 ]
  7. Pastores GM: Miglustat: substrate reduction therapy for lysosomal storage disorders associated with primary central nervous system involvement. Recent Pat CNS Drug Discov. 2006 Jan;1(1):77-82. [PubMed:18221193 ]
  8. McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. [PubMed:14609352 ]
  9. Giraldo P, Latre P, Alfonso P, Acedo A, Alonso D, Barez A, Corrales A, Franco R, Roldan V, Serrano S, Pocovi M: Short-term effect of miglustat in every day clinical use in treatment-naive or previously treated patients with type 1 Gaucher's disease. Haematologica. 2006 May;91(5):703-6. Epub 2006 Apr 19. [PubMed:16627252 ]
  10. Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352 ]