VENGLUSTAT HYDROCHLORIDE

Investigational

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C20H24FN3O2S.ClH
Molecular Weight	425.948
Optical Activity	UNSPECIFIED
Defined Stereocenters	1 / 1
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

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

InChI

InChIKey=OTUWNTGDKASFHL-PKLMIRHRSA-N

InChI=1S/C20H24FN3O2S.ClH/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);1H/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

Molecular Formula	ClH
Molecular Weight	36.461
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/26948439 | https://www.ncbi.nlm.nih.gov/pubmed/25938659 | http://adisinsight.springer.com/drugs/800036945

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

Targets

Primary Target	Pharmacology	Condition	Potency
Ceramide glucosyltransferase 9 Target ID: CHEMBL2063 Sources: http://adisinsight.springer.com/drugs/800036945	Inhibitor 8504

Conditions

Condition	Modality	Highest Phase	Product
Parkinson's disease 232 Sources: http://adisinsight.springer.com/drugs/800036945	Primary	Phase II 1147	Unknown Approved Use Unknown
Gaucher's disease type III 7 Sources: https://www.ncbi.nlm.nih.gov/pubmed/26948439	Primary	Phase II 1147	Unknown Approved Use Unknown
Fabry's disease 6 Sources: https://www.ncbi.nlm.nih.gov/pubmed/25938659	Primary	Phase II 1147	Unknown Approved Use Unknown

C_max

Value	Dose	Analyte	Population
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
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

AUC

Value	Dose	Analyte	Population
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
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

T_1/2

Value	Dose	Co-administered	Analyte	Population
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

CYP3A4	CYP2C9	CYP2D6	hERG
substrate 1946

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
P-gp 1741

Drug as perpetrator

Target	Modality	Activity	Metabolite	Clinical evidence
P-gp 1932	inhibitor 4027 Sources: https://pubmed.ncbi.nlm.nih.gov/32851809/	yes

Drug as victim

Target	Modality	Activity	Metabolite	Clinical evidence
CYP3A4 1946	substrate 4014 Sources: https://pubmed.ncbi.nlm.nih.gov/32851809/	major

Sourcing

Vendor/Aggregator	ID	URL
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
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
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
mcule	MCULE-6327706056	https://mcule.com/MCULE-6327706056/
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=
Molnova	M11673	https://www.molnova.com/en/serch-list.html?keywords=M11673
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
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

PubMed

Title	Date	PubMed
CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease.	2016-06	26948439
Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease.	2015-04-30	25938659

Patents

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 `Mon Mar 31 18:14:20 GMT 2025` Edited by `admin` on `Mon Mar 31 18:14:20 GMT 2025`
Record UNII	OK1S17555O
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

IBIGLUSTAT HYDROCHLORIDE

Preferred Name

English

VENGLUSTAT HYDROCHLORIDE

Common Name

English

CARBAMIC ACID, N-(1-(2-(4-FLUOROPHENYL)-4-THIAZOLYL)-1-METHYLETHYL)-, (3S)-1-AZABICYCLO(2.2.2)OCT-3-YL ESTER, HYDROCHLORIDE (1:1)

Systematic Name

English

Code System Code Type Description

FDA UNII

OK1S17555O