You are using an unsupported browser. Please upgrade your browser to a newer version to get the best experience on DrugBank.
Identification
NameMiglustat
Accession NumberDB00419  (APRD01118)
TypeSmall Molecule
GroupsApproved
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. Miglustat 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. 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.
Structure
Thumb
Synonyms
BuDNJ
Butyldeoxynojirimycin
Miglustatum
N-(N-Butyl)deoxynojirimycin
N-Butyl deoxynojirimycin
N-Butyl-1-deoxynojirimycin
N-Butylmoranoline
NB-dnj
SC-48334
Zavesca
External Identifiers Not Available
Approved Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
Sandoz MiglustatCapsule100 mgOralSandoz Canada IncorporatedNot applicableNot applicableCanada
ZavescaCapsule100 mgOralActelion Pharmaceuticals Ltd2004-05-26Not applicableCanada
ZavescaCapsule100 mgOralActelion Ltd2002-11-20Not applicableEu
ZavescaCapsule100 mg/1OralActelion Pharmaceuticals US, Inc.2003-07-31Not applicableUs
Approved Generic Prescription ProductsNot Available
Approved Over the Counter ProductsNot Available
Unapproved/Other Products Not Available
International BrandsNot Available
Brand mixturesNot Available
Salts
Name/CASStructureProperties
Miglustat Hydrochloride
Thumb
  • InChI Key: UQRORFVVSGFNRO-UTINFBMNSA-N
  • Monoisotopic Mass: 219.147058165
  • Average Mass: 219.278
DBSALT000621
Categories
UNIIADN3S497AZ
CAS number72599-27-0
WeightAverage: 219.278
Monoisotopic: 219.147058165
Chemical FormulaC10H21NO4
InChI KeyUQRORFVVSGFNRO-UTINFBMNSA-N
InChI
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
IUPAC Name
(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
SMILES
CCCCN1C[[email protected]](O)[C@@H](O)[[email protected]](O)[[email protected]]1CO
Pharmacology
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).
Structured Indications
PharmacodynamicsMiglustat, an N-alkylated imino sugar, is a synthetic analogue of D-glucose. Miglustat is an inhibitor of the enzyme glucosylceramide synthase, which is a glucosyl transferase enzyme responsible for catalyzing the formation of glucosylceramide (glucocerebroside). Glucosylceramide is a substrate for the endogenous glucocerebrosidase, an enzyme that is deficient in Gaucher's disease. The accumulation of glucosylceramide due to the absence of glucocerebrosidase results in the storage of this material in the lysosomes of tissue macrophages, leading to widespread pathology due to infiltration of lipid-engorged macrophages in the viscera, lymph nodes, and bone marrow. This results in secondary hematologic consequences including sever anemia and thrombocytopenia, in addition to the characteristic progressive hepatosplenomegaly, as well as skeletal complications including osteonecrosis and osteopenia with secondary pathological fractures.
Mechanism of actionMiglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. The goal of treatment with miglustat is to reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy), reducing the accumulation of glucocerebroside in macrophages. In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids. In clinical trials, miglustat improved liver and spleen volume, as well as hemoglobin concentration and platelet count. Inhibition of glycosphingolipid synthesis has also shown to reduce intracellular lipid storage, improve fluid-phase endosomal uptake and normalize lipid transport in peripheral blood B lymphocytes of NP-C patients, which results in a decrease in the potentially neurotoxic accumulation of gnagliosides GM2 and GM3, lactosylceramide and glucosylceramide, possibly preventing further neuronal damage. Other studies have also suggested that miglustat may indirectly modulate intracellular calcium homeostasis through its effects on glucosylceramide levels, and evidence has shown that an initiating factor in the pathogenesis of NP-C may be impaired calcium homeostasis related to sphingosine storage. Therefore, the effect that miglustat exerts on intracellular calcium levels may influence an important underlying pathogenic mechanism of NP-C.
TargetKindPharmacological actionActionsOrganismUniProt ID
Ceramide glucosyltransferaseProteinyes
inhibitor
HumanQ16739 details
Related Articles
AbsorptionMean oral bioavailability is 97%.
Volume of distributionNot Available
Protein bindingMiglustat does not bind to plasma proteins.
Metabolism

There is no evidence that miglustat is metabolized in humans.

Route of eliminationNot Available
Half lifeThe effective half-life of miglustat is approximately 6 to 7 hours.
ClearanceNot Available
ToxicityMiglustat has been administered at doses of up to 3000 mg/day (approximately 10 times the recommended starting dose administered to Gaucher patients) for up to six months in Human Immunodeficiency Virus (HIV)-positive patients. Adverse events observed in the HIV studies included granulocytopenia, dizziness, and paresthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or above.
Affected organisms
  • Humans and other mammals
PathwaysNot Available
SNP Mediated EffectsNot Available
SNP Mediated Adverse Drug ReactionsNot Available
Interactions
Drug Interactions
DrugInteractionDrug group
AripiprazoleThe therapeutic efficacy of Miglustat can be decreased when used in combination with Aripiprazole.Approved, Investigational
Arsenic trioxideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Arsenic trioxide.Approved, Investigational
ArticaineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Articaine.Approved
AsenapineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Asenapine.Approved
AtazanavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Atazanavir.Approved, Investigational
BendroflumethiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Bendroflumethiazide.Approved
BetamethasoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Betamethasone.Approved, Vet Approved
BrexpiprazoleThe therapeutic efficacy of Miglustat can be decreased when used in combination with Brexpiprazole.Approved
BumetanideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Bumetanide.Approved
BuserelinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Buserelin.Approved
CeritinibThe therapeutic efficacy of Miglustat can be decreased when used in combination with Ceritinib.Approved
ChlorothiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Chlorothiazide.Approved, Vet Approved
ChlorpropamideMiglustat may increase the hypoglycemic activities of Chlorpropamide.Approved
ChlorthalidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Chlorthalidone.Approved
ClozapineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Clozapine.Approved
CorticotropinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Corticotropin.Approved, Vet Approved
Cortisone acetateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Cortisone acetate.Approved
Cyproterone acetateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Cyproterone acetate.Approved, Investigational
DabrafenibThe therapeutic efficacy of Miglustat can be decreased when used in combination with Dabrafenib.Approved
DanazolThe therapeutic efficacy of Miglustat can be decreased when used in combination with Danazol.Approved
DarunavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Darunavir.Approved
DesogestrelThe therapeutic efficacy of Miglustat can be decreased when used in combination with Desogestrel.Approved
DexamethasoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Dexamethasone.Approved, Investigational, Vet Approved
DiazoxideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Diazoxide.Approved
DienogestThe therapeutic efficacy of Miglustat can be decreased when used in combination with Dienogest.Approved
DisopyramideMiglustat may increase the hypoglycemic activities of Disopyramide.Approved
DrospirenoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Drospirenone.Approved
EpinephrineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Epinephrine.Approved, Vet Approved
EstradiolThe therapeutic efficacy of Miglustat can be decreased when used in combination with Estradiol.Approved, Investigational, Vet Approved
Estrone sulfateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Estrone sulfate.Approved
Etacrynic acidThe therapeutic efficacy of Miglustat can be decreased when used in combination with Etacrynic acid.Approved
Ethinyl EstradiolThe therapeutic efficacy of Miglustat can be decreased when used in combination with Ethinyl Estradiol.Approved
Ethynodiol diacetateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Ethynodiol diacetate.Approved
EtonogestrelThe therapeutic efficacy of Miglustat can be decreased when used in combination with Etonogestrel.Approved, Investigational
EverolimusThe therapeutic efficacy of Miglustat can be decreased when used in combination with Everolimus.Approved
FludrocortisoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Fludrocortisone.Approved
FosamprenavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Fosamprenavir.Approved
FurosemideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Furosemide.Approved, Vet Approved
GliclazideMiglustat may increase the hypoglycemic activities of Gliclazide.Approved
GlimepirideMiglustat may increase the hypoglycemic activities of Glimepiride.Approved
GlipizideMiglustat may increase the hypoglycemic activities of Glipizide.Approved
GlyburideMiglustat may increase the hypoglycemic activities of Glyburide.Approved
GoserelinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Goserelin.Approved
HistrelinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Histrelin.Approved
HydrochlorothiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Hydrochlorothiazide.Approved, Vet Approved
HydrocortisoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Hydrocortisone.Approved, Vet Approved
HydroflumethiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Hydroflumethiazide.Approved
Hydroxyprogesterone caproateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Hydroxyprogesterone caproate.Approved
IloperidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Iloperidone.Approved
IndapamideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Indapamide.Approved
IndinavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Indinavir.Approved
Insulin AspartMiglustat may increase the hypoglycemic activities of Insulin Aspart.Approved
Insulin DetemirMiglustat may increase the hypoglycemic activities of Insulin Detemir.Approved
Insulin GlargineMiglustat may increase the hypoglycemic activities of Insulin Glargine.Approved
Insulin GlulisineMiglustat may increase the hypoglycemic activities of Insulin Glulisine.Approved
Insulin HumanMiglustat may increase the hypoglycemic activities of Insulin Human.Approved, Investigational
Insulin LisproMiglustat may increase the hypoglycemic activities of Insulin Lispro.Approved
LanreotideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Lanreotide.Approved
LeuprolideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Leuprolide.Approved, Investigational
LevonorgestrelThe therapeutic efficacy of Miglustat can be decreased when used in combination with Levonorgestrel.Approved, Investigational
Lipoic AcidLipoic Acid may increase the hypoglycemic activities of Miglustat.Approved, Nutraceutical
LopinavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Lopinavir.Approved
LurasidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Lurasidone.Approved
MecaserminMiglustat may increase the hypoglycemic activities of Mecasermin.Approved, Investigational
Medroxyprogesterone acetateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Medroxyprogesterone acetate.Approved, Investigational
Megestrol acetateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Megestrol acetate.Approved, Vet Approved
MestranolThe therapeutic efficacy of Miglustat can be decreased when used in combination with Mestranol.Approved
MethotrimeprazineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Methotrimeprazine.Approved
MethyclothiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Methyclothiazide.Approved
MethylprednisoloneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Methylprednisolone.Approved, Vet Approved
MetolazoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Metolazone.Approved
MifepristoneMiglustat may increase the hypoglycemic activities of Mifepristone.Approved, Investigational
NateglinideMiglustat may increase the hypoglycemic activities of Nateglinide.Approved, Investigational
NelfinavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Nelfinavir.Approved
NiacinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Niacin.Approved, Investigational, Nutraceutical
NilotinibThe therapeutic efficacy of Miglustat can be decreased when used in combination with Nilotinib.Approved, Investigational
NorethisteroneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Norethisterone.Approved
NorgestimateThe therapeutic efficacy of Miglustat can be decreased when used in combination with Norgestimate.Approved
OctreotideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Octreotide.Approved, Investigational
OlanzapineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Olanzapine.Approved, Investigational
PaliperidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Paliperidone.Approved
PasireotideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Pasireotide.Approved
PentamidineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Pentamidine.Approved
PiperazineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Piperazine.Approved, Vet Approved
PipotiazineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Pipotiazine.Approved
PolythiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Polythiazide.Approved
PrednisoloneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Prednisolone.Approved, Vet Approved
PrednisoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Prednisone.Approved, Vet Approved
ProgesteroneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Progesterone.Approved, Vet Approved
QuetiapineThe therapeutic efficacy of Miglustat can be decreased when used in combination with Quetiapine.Approved
QuinethazoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Quinethazone.Approved
QuinineMiglustat may increase the hypoglycemic activities of Quinine.Approved
RepaglinideMiglustat may increase the hypoglycemic activities of Repaglinide.Approved, Investigational
RisperidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Risperidone.Approved, Investigational
RitonavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Ritonavir.Approved, Investigational
SaquinavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Saquinavir.Approved, Investigational
SirolimusThe therapeutic efficacy of Miglustat can be decreased when used in combination with Sirolimus.Approved, Investigational
SulfadiazineMiglustat may increase the hypoglycemic activities of Sulfadiazine.Approved, Vet Approved
SulfamethoxazoleMiglustat may increase the hypoglycemic activities of Sulfamethoxazole.Approved
SulfisoxazoleMiglustat may increase the hypoglycemic activities of Sulfisoxazole.Approved, Vet Approved
SunitinibMiglustat may increase the hypoglycemic activities of Sunitinib.Approved, Investigational
TacrolimusThe therapeutic efficacy of Miglustat can be decreased when used in combination with Tacrolimus.Approved, Investigational
TemsirolimusThe therapeutic efficacy of Miglustat can be decreased when used in combination with Temsirolimus.Approved
TipranavirThe therapeutic efficacy of Miglustat can be decreased when used in combination with Tipranavir.Approved, Investigational
TolazamideMiglustat may increase the hypoglycemic activities of Tolazamide.Approved
TolbutamideMiglustat may increase the hypoglycemic activities of Tolbutamide.Approved
TorasemideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Torasemide.Approved
TriamcinoloneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Triamcinolone.Approved, Vet Approved
TrichlormethiazideThe therapeutic efficacy of Miglustat can be decreased when used in combination with Trichlormethiazide.Approved, Vet Approved
TriptorelinThe therapeutic efficacy of Miglustat can be decreased when used in combination with Triptorelin.Approved, Vet Approved
VorinostatThe therapeutic efficacy of Miglustat can be decreased when used in combination with Vorinostat.Approved, Investigational
ZiprasidoneThe therapeutic efficacy of Miglustat can be decreased when used in combination with Ziprasidone.Approved
Food Interactions
  • Take without regard to meals, but always at the same time and in the same way.
References
Synthesis ReferenceNot Available
General References
  1. 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 ]
  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. 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 ]
  4. 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 ]
  5. 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 ]
  6. McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. [PubMed:14609352 ]
External Links
ATC CodesA16AX06
AHFS Codes
  • 92:00.00
PDB EntriesNot Available
FDA labelDownload (911 KB)
MSDSNot Available
ADMET
Predicted ADMET features
PropertyValueProbability
Human Intestinal Absorption+0.9092
Blood Brain Barrier-0.9011
Caco-2 permeable-0.6433
P-glycoprotein substrateSubstrate0.8088
P-glycoprotein inhibitor INon-inhibitor0.667
P-glycoprotein inhibitor IINon-inhibitor0.9556
Renal organic cation transporterNon-inhibitor0.757
CYP450 2C9 substrateNon-substrate0.8224
CYP450 2D6 substrateNon-substrate0.765
CYP450 3A4 substrateNon-substrate0.5817
CYP450 1A2 substrateNon-inhibitor0.9188
CYP450 2C9 inhibitorNon-inhibitor0.8855
CYP450 2D6 inhibitorNon-inhibitor0.9226
CYP450 2C19 inhibitorNon-inhibitor0.9484
CYP450 3A4 inhibitorNon-inhibitor0.9855
CYP450 inhibitory promiscuityLow CYP Inhibitory Promiscuity0.9941
Ames testNon AMES toxic0.8426
CarcinogenicityNon-carcinogens0.9595
BiodegradationNot ready biodegradable0.5763
Rat acute toxicity2.1203 LD50, mol/kg Not applicable
hERG inhibition (predictor I)Weak inhibitor0.6675
hERG inhibition (predictor II)Non-inhibitor0.8508
ADMET data is predicted using admetSAR, a free tool for evaluating chemical ADMET properties. (23092397 )
Pharmacoeconomics
Manufacturers
  • Actelion pharmaceuticals ltd
Packagers
Dosage forms
FormRouteStrength
CapsuleOral100 mg
CapsuleOral100 mg/1
Prices
Unit descriptionCostUnit
Zavesca 100 mg capsule160.67USD capsule
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)
US5472969 No1993-05-132013-05-13Us
US5525616 No1993-06-112013-06-11Us
Properties
StateSolid
Experimental Properties
PropertyValueSource
melting point169-172 °CNot Available
water solubilityHighly soluble in water (>1000 mg/mL as a free base).Not Available
logP-0.6Not Available
Predicted Properties
PropertyValueSource
Water Solubility331.0 mg/mLALOGPS
logP-1.1ALOGPS
logP-1.2ChemAxon
logS0.18ALOGPS
pKa (Strongest Acidic)12.9ChemAxon
pKa (Strongest Basic)8.49ChemAxon
Physiological Charge1ChemAxon
Hydrogen Acceptor Count5ChemAxon
Hydrogen Donor Count4ChemAxon
Polar Surface Area84.16 Å2ChemAxon
Rotatable Bond Count4ChemAxon
Refractivity55.74 m3·mol-1ChemAxon
Polarizability23.99 Å3ChemAxon
Number of Rings1ChemAxon
Bioavailability1ChemAxon
Rule of FiveYesChemAxon
Ghose FilterYesChemAxon
Veber's RuleYesChemAxon
MDDR-like RuleYesChemAxon
Spectra
Mass Spec (NIST)Not Available
Spectra
Spectrum TypeDescriptionSplash Key
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 10V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 20V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 40V, PositiveNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 10V, NegativeNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 20V, NegativeNot Available
Predicted LC-MS/MSPredicted LC-MS/MS Spectrum - 40V, NegativeNot Available
Taxonomy
DescriptionThis compound belongs to the class of organic compounds known as piperidines. These 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
  • Tertiary aliphatic amine
  • Tertiary amine
  • Secondary alcohol
  • Polyol
  • 1,2-diol
  • 1,2-aminoalcohol
  • Azacycle
  • Hydrocarbon derivative
  • Primary alcohol
  • Organooxygen compound
  • Organonitrogen compound
  • Amine
  • Alcohol
  • Aliphatic heteromonocyclic compound
Molecular FrameworkAliphatic heteromonocyclic compounds
External Descriptors

Targets

Kind
Protein
Organism
Human
Pharmacological action
yes
Actions
inhibitor
General Function:
Ceramide glucosyltransferase activity
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 Da
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 ]
Comments
comments powered by Disqus
Drug created on June 13, 2005 07:24 / Updated on December 08, 2016 11:11