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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

Description

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.

CNS Activity

Originator

Approval Year

Unknown
139594
Targets

Targets

Primary TargetPharmacologyConditionPotency
Inhibitor
8297
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Phase II
1132
Unknown

Approved Use

Unknown
Primary
Phase II
1132
Unknown

Approved Use

Unknown
Primary
Phase II
1132
Unknown

Approved Use

Unknown
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
68 ng/mL
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
EXPERIMENT
https://pubmed.ncbi.nlm.nih.gov/32851809
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
substrate
1737

OverviewOther

Other InhibitorOther SubstrateOther Inducer
P-gp
1461

Major CYP
4
Major CYP
4
Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
Major CYP
4
 yes
Major CYP
4
 yes
P-gp
1650
 yes
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
CYP3A4
1737
 major
Sourcing

Sourcing

Vendor/AggregatorIDURL
1PlusChem LLC
1P001CHT
https://www.1pchem.com/search?query=1P001CHT
1PlusChem LLC
1P01EOHK
https://www.1pchem.com/search?query=1P01EOHK
AChemBlock
M15692
http://www.achemblock.com/catalogsearch/result/?q=M15692
AstaTech
P14969
http://astatechinc.com/CPSResult.php?CRNO=P14969
BLD Pharm
BD445977
http://www.bldpharm.com/search/Search.html?keyword=BD445977
Chem Scene
CS-0069906
http://www.chemscene.com/goproductList/searchProductList?productObj=CS-0069906
Chem Scene
CS-6512
http://www.chemscene.com/goproductList/searchProductList?productObj=CS-6512
ChemShuttle
186455
https://www.chemshuttle.com/catalogsearch/result/?q=186455
Fluorochem
504966
http://www.fluorochem.co.uk/Products/Product?code=504966
KeyOrganics
AS-52367
http://www.keyorganicsinc.com/bionet/catalogsearch/result?q=AS-52367
Lab Network
LN01344120
https://labnetwork.com/frontend-app/p/#!/moleculedetails/LN01344120
MedChem Express
HY-16743
https://www.medchemexpress.com/search.html?q=HY-16743A&ft=&fa=&fp=
MedChem Express
HY-16743A
https://www.medchemexpress.com/search.html?q=HY-16743A&ft=&fa=&fp=
MolPort
MolPort-042-681-312
https://www.molport.com/shop/molecule-link/MolPort-042-681-312
MolPort
MolPort-046-683-427
https://www.molport.com/shop/molecule-link/MolPort-046-683-427
Molnova
M11673
https://www.molnova.com/en/serch-list.html?keywords=M11673
MuseChem
I007242
https://www.musechem.com/product/I007242.html
ShangHai Biochempartner
BCP20857
http://www.biochempartner.com/search_BCP20857
TargetMol
T4473
https://www.targetmol.com/search?keyword=T4473
eMolecules
168789254
https://orderbb.emolecules.com/search/#?query=168789254
eMolecules
302090446
https://orderbb.emolecules.com/search/#?query=302090446
eMolecules
56974539
https://orderbb.emolecules.com/search/#?query=56974539
eNovation Chemicals
D572887
https://www.enovationchem.com/Products.asp?SEARCHTEXT=D572887&searchbtn.x=0&searchbtn.y=0
mcule
MCULE-6327706056
https://mcule.com/MCULE-6327706056/
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

Patents

20140255381
4
20140371460
4
9126993
4
9139580
4
WO2012129084A2
4
WO2015089067A1
4

Sample Use Guides

In Vivo Use Guide
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 GMT 2023
Edited
by admin
on Sat Dec 16 16:16:06 GMT 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 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
FDA ORPHAN DRUG 439814
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
EU-Orphan Drug EU/3/18/2122
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
FDA ORPHAN DRUG 637218
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
NCI_THESAURUS C471
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
Code System Code Type Description
FDA UNII
BLP1XA3FZA
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
CAS
1401090-53-6
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
USAN
GH-116
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
INN
10063
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
DRUG BANK
DB14966
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
PUBCHEM
60199242
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
NCI_THESAURUS
C152852
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
SMS_ID
100000177429
Created by admin on Sat Dec 16 16:16:07 GMT 2023 , Edited by admin on Sat Dec 16 16:16:07 GMT 2023
PRIMARY
Related Record Type Details
Structure of VENGLUSTAT SUCCINATE
SALT/SOLVATE -> PARENT
Structure of VENGLUSTAT HYDROCHLORIDE
SALT/SOLVATE -> PARENT
CERAMIDE GLUCOSYLTRANSFERASE
TARGET -> INHIBITOR
Structure of VENGLUSTAT MALATE
SALT/SOLVATE -> PARENT
Related Record Type Details
Structure of VENGLUSTAT
ACTIVE MOIETY
NIH
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