Details of the Drug

General Information of Drug (ID: DM5J64S)

Drug Name
Miglustat
Synonyms
BuDNJ; Butyldeoxynojirimycin; Miglustatum; NBV; Vevesca; Zavesca; Miglustat [USAN]; OGT 918; SC 48334; SC48334; Miglustat, Hydrochloride; N-Butyl deoxynojirimycin; N-Butyl dnj; N-Butyldeoxynojirimycin; N-Butylmoranoline; NB-DNJ; OGT-918; SC-48334; Zavesca (TN); Miglustat (USAN/INN); N-Bu-DNJ; N-Butyl-DNJ; N-Butyl-deoxynojirimycin; N-(n-Butyl)deoxynojirimycin; N-butyl-1-deoxynojirimycin; N-(n-butyl)-1,5-dideoxy-1,5-imino-D-glucitol; (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol; 1,5-Dideoxy-1,5-N-butylimino-D-glucitol
Indication
Disease Entry ICD 11 Status REF
Niemann-Pick disease type C N.A. Approved [1]
Type 1 gaucher disease 5C56.0Y Approved [2]
Gaucher disease 5C56.0Y Investigative [1]
Therapeutic Class
Anti-HIV Agents
Drug Type
Small molecular drug
Structure
3D MOL 2D MOL
#Ro5 Violations (Lipinski): 0 Molecular Weight (mw) 219.28
Logarithm of the Partition Coefficient (xlogp) -0.6
Rotatable Bond Count (rotbonds) 4
Hydrogen Bond Donor Count (hbonddonor) 4
Hydrogen Bond Acceptor Count (hbondacc) 5
ADMET Property
BDDCS Class
Biopharmaceutics Drug Disposition Classification System (BDDCS) Class 3: high solubility and low permeability [3]
Bioavailability
The bioavailability of drug is 97% []
Elimination
85% of drug is excreted from urine in the unchanged form [3]
Half-life
The concentration or amount of drug in body reduced by one-half in 6 - 7 hours [4]
MRTD
The Maximum Recommended Therapeutic Dose (MRTD) of drug that ensured maximising efficacy and moderate side effect is 19.544 micromolar/kg/day [5]
Water Solubility
The ability of drug to dissolve in water is measured as 1000 mg/mL [3]
Chemical Identifiers
Formula
C10H21NO4
IUPAC Name
(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
Canonical SMILES
CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O
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
InChIKey
UQRORFVVSGFNRO-UTINFBMNSA-N
Cross-matching ID
PubChem CID
51634
ChEBI ID
CHEBI:50381
CAS Number
72599-27-0
DrugBank ID
DB00419
TTD ID
D0HR8Z
ACDINA ID
D00434
Combinatorial Drugs (CBD) Click to Jump to the Detailed CBD Information of This Drug
Repurposed Drugs (RPD) Click to Jump to the Detailed RPD Information of This Drug

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Ceramide glucosyltransferase (UGCG) TTPHEX3 CEGT_HUMAN Inhibitor [6]

Drug Off-Target (DOT)
DOT Name DOT ID UniProt ID Interaction REF
Cystic fibrosis transmembrane conductance regulator (CFTR) OT6B22QH CFTR_HUMAN Gene/Protein Processing [7]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

Drug Inactive Ingredient(s) (DIG) and Formulation(s) of This Drug

DIG
DIG Name DIG ID PubChem CID Functional Classification
Ferrosoferric oxide E00231 14789 Colorant
Magnesium stearate E00208 11177 lubricant
Titanium dioxide E00322 26042 Coating agent; Colorant; Opacifying agent
Vinylpyrrolidone E00668 Not Available Binding agent; Coating agent; Disintegrant; Film/membrane-forming agent; Solubilizing agent; Suspending agent
Pharmaceutical Formulation
Formulation Name Drug Dosage Dosage Form Route
Miglustat 100 mg capsule 100 mg Oral Capsule Oral
Jump to Detail Pharmaceutical Formulation Page of This Drug

References

1 Miglustat FDA Label
2 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 4841).
3 BDDCS applied to over 900 drugs
4 Trend Analysis of a Database of Intravenous Pharmacokinetic Parameters in Humans for 1352 Drug Compounds
5 Estimating the safe starting dose in phase I clinical trials and no observed effect level based on QSAR modeling of the human maximum recommended daily dose
6 Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Clin Ther. 2007 Aug;29(8):1645-54.
7 Proteasome-dependent pharmacological rescue of cystic fibrosis transmembrane conductance regulator revealed by mutation of glycine 622. J Pharmacol Exp Ther. 2008 Apr;325(1):89-99. doi: 10.1124/jpet.107.134502. Epub 2008 Jan 29.