U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ABSOLUTE
Molecular Formula C20H24FN3O2S
Molecular Weight 389.487
Optical Activity UNSPECIFIED
Defined Stereocenters 1 / 1
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of VENGLUSTAT

SMILES

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

InChI

InChIKey=YFHRCLAKZBDRHN-MRXNPFEDSA-N
InChI=1S/C20H24FN3O2S/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/h3-6,12-13,16H,7-11H2,1-2H3,(H,23,25)/t16-/m1/s1

HIDE SMILES / InChI

Molecular Formula C20H24FN3O2S
Molecular Weight 389.487
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 1 / 1
E/Z Centers 0
Optical Activity UNSPECIFIED

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
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
Substance Class Chemical
Created
by admin
on Sat Dec 16 16:16:06 UTC 2023
Edited
by admin
on Sat Dec 16 16:16:06 UTC 2023
Record UNII
BLP1XA3FZA
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
VENGLUSTAT
INN   WHO-DD  
INN   USAN  
Official Name English
GZ402671
Code English
GZ/SAR402671
Code English
venglustat [INN]
Common Name English
VENGLUSTAT [USAN]
Common Name English
(3S)-1-Azabicyclo[2.2.2]octan-3-yl N-{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Systematic Name English
GZ-402671
Code English
SAR-402671
Code English
GZ/SAR-402671
Code English
IBIGLUSTAT
INN  
Official Name English
GENZ-682452-AA
Code English
Venglustat [WHO-DD]
Common Name English
GENZ-682452
Code English
SAR402671
Common Name English
CARBAMIC ACID, N-(1-(2-(4-FLUOROPHENYL)-4-THIAZOLYL)-1-METHYLETHYL)-, (3S)-1-AZABICYCLO(2.2.2)OCT-3-YL ESTER
Systematic Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 443414
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
FDA ORPHAN DRUG 439814
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
EU-Orphan Drug EU/3/18/2122
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
FDA ORPHAN DRUG 637218
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
NCI_THESAURUS C471
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
Code System Code Type Description
FDA UNII
BLP1XA3FZA
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
CAS
1401090-53-6
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
USAN
GH-116
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
INN
10063
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
DRUG BANK
DB14966
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
PUBCHEM
60199242
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
NCI_THESAURUS
C152852
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
SMS_ID
100000177429
Created by admin on Sat Dec 16 16:16:07 UTC 2023 , Edited by admin on Sat Dec 16 16:16:07 UTC 2023
PRIMARY
Related Record Type Details
SALT/SOLVATE -> PARENT
SALT/SOLVATE -> PARENT
TARGET -> INHIBITOR
SALT/SOLVATE -> PARENT
Related Record Type Details
ACTIVE MOIETY