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Details

Stereochemistry ABSOLUTE
Molecular Formula C20H24FN3O2S.C4H6O5
Molecular Weight 523.574
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of VENGLUSTAT MALATE

SMILES

O[C@@H](CC(O)=O)C(O)=O.CC(C)(NC(=O)O[C@@H]1CN2CCC1CC2)C3=CSC(=N3)C4=CC=C(F)C=C4

InChI

InChIKey=SQXUKOJKIWCALK-AAXLQGCPSA-N
InChI=1S/C20H24FN3O2S.C4H6O5/c1-20(2,17-12-27-18(22-17)14-3-5-15(21)6-4-14)23-19(25)26-16-11-24-9-7-13(16)8-10-24;5-2(4(8)9)1-3(6)7/h3-6,12-13,16H,7-11H2,1-2H3,(H,23,25);2,5H,1H2,(H,6,7)(H,8,9)/t16-;2-/m10/s1

HIDE SMILES / InChI
Ibiglustat (GZ/SAR402671 or Genz-682452) is a small molecule inhibitor of glucosylceramide synthase. Ibiglustat has been demonstrated to effectively lower glycosphingolipid synthesis. Genzyme, a Sanofi Company is developing Ibiglustat for the treatment of Parkinson's Disease, Gaucher Disease, and Fabry Disease.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
Primary
Unknown

Approved Use

Unknown
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
68 ng/mL
20 mg single, oral
dose: 20 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
142 ng/mL
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
529 ng/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
1100 ng × h/mL
20 mg single, oral
dose: 20 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
2420 ng × h/mL
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
20600 ng × h/mL
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
35 h
20 mg 1 times / day steady-state, oral
dose: 20 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
26.9 h
150 mg single, oral
dose: 150 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VENGLUSTAT plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
Overview

Overview

CYP3A4CYP2C9CYP2D6hERG

OverviewOther

Other InhibitorOther SubstrateOther Inducer



Drug as perpetrator​Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
major
Sourcing

Sourcing

Vendor/AggregatorIDURL
PubMed

PubMed

TitleDatePubMed
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.
2015 Apr 30
CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.
2016 Jun
Patents

Sample Use Guides

A phase 2 study assessed the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of Ibiglustat (GZ/SAR402671) in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease: GZ/SAR402671 15 mg once daily orally for 26 weeks.
Route of Administration: Oral
In Vitro Use Guide
Unknown
Name Type Language
VENGLUSTAT MALATE
USAN  
Official Name English
GZ/SAR-402671A
Code English
GZ/SAR402671A
Code English
VENGLUSTAT MALATE [USAN]
Common Name English
BUTANEDIOIC ACID, 2-HYDROXY-, (2S)-, COMPD. WITH (3S)-1-AZABICYCLO(2.2.2)OCT-3-YL N-(1-(2-(4-FLUOROPHENYL)-4-THIAZOLYL)-1-METHYLETHYL)CARBAMATE (1:1)
Systematic Name English
VENGLUSTAT L-MALATE
Common Name English
(2S)-2-HYDROXYBUTANEDIOIC ACID - (3S)-1- AZABICYCLO(2.2.2)OCT-3-YL (2-(2-(4-FLUOROPHENYL)-1,3-THIAZOL-4-YL)PROPAN-2-YL) CARBAMATE
Systematic Name English
IBIGLUSTAT L-MALATE
Common Name English
SAR-402671 L-MALATE
Code English
GENZ-682452-AU
Code English
SAR402671A
Code English
SAR-402671A
Code English
Classification Tree Code System Code
FDA ORPHAN DRUG 755820
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
Code System Code Type Description
USAN
GH-117
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
EVMPD
SUB192872
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
SMS_ID
100000177469
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
NCI_THESAURUS
C171783
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
CAS
1629063-78-0
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
FDA UNII
94855LQZ8D
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY
PUBCHEM
60199241
Created by admin on Fri Dec 15 15:55:39 GMT 2023 , Edited by admin on Fri Dec 15 15:55:39 GMT 2023
PRIMARY