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
Type 1 gaucher disease	5C56.0Y	Approved	[1], [2]

Therapeutic Class

Anti-HIV Agents

Drug Type

Small molecular drug

Structure

3D MOL

2D MOL

#Ro5 Violations (Lipinski): 0

Molecular Weight (mw)

219.28

Topological Polar Surface Area (xlogp)

-0.6

Rotatable Bond Count (rotbonds)

Hydrogen Bond Donor Count (hbonddonor)

Hydrogen Bond Acceptor Count (hbondacc)

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% [4]
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 [5]
MRTD: The Maximum Recommended Therapeutic Dose (MRTD) of drug that ensured maximising efficacy and moderate side effect is 19.544 micromolar/kg/day [6]
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

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	[7]

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Molecular Interaction Atlas (MIA)

MIA Detail

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
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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	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).
2	Natural products as sources of new drugs over the last 25 years. J Nat Prod. 2007 Mar;70(3):461-77.
3	BDDCS applied to over 900 drugs
4	FDA approval: ado-trastuzumab emtansine for the treatment of patients with HER2-positive metastatic breast cancer. Clin Cancer Res. 2014 Sep 1;20(17):4436-41.
5	Trend Analysis of a Database of Intravenous Pharmacokinetic Parameters in Humans for 1352 Drug Compounds
6	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
7	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.
8	2014 FDA drug approvals. Nat Rev Drug Discov. 2015 Feb;14(2):77-81.
9	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
10	Accumulation of glucosylceramide in murine testis, caused by inhibition of beta-glucosidase 2: implications for spermatogenesis. J Biol Chem. 2007 Nov 9;282(45):32655-64.