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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

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

Targets

Primary TargetPharmacologyConditionPotency

Conditions

ConditionModalityTargetsHighest PhaseProduct
Primary
Unknown
Primary
Unknown
Primary
Unknown

Cmax

ValueDoseCo-administeredAnalytePopulation
142 ng/mL
20 mg 1 times / day steady-state, oral
VENGLUSTAT plasma
Homo sapiens
529 ng/mL
150 mg single, oral
VENGLUSTAT plasma
Homo sapiens
68 ng/mL
20 mg single, oral
VENGLUSTAT plasma
Homo sapiens

AUC

ValueDoseCo-administeredAnalytePopulation
2420 ng × h/mL
20 mg 1 times / day steady-state, oral
VENGLUSTAT plasma
Homo sapiens
20600 ng × h/mL
150 mg single, oral
VENGLUSTAT plasma
Homo sapiens
1100 ng × h/mL
20 mg single, oral
VENGLUSTAT plasma
Homo sapiens

T1/2

ValueDoseCo-administeredAnalytePopulation
35 h
20 mg 1 times / day steady-state, oral
VENGLUSTAT plasma
Homo sapiens
26.9 h
150 mg single, oral
VENGLUSTAT plasma
Homo sapiens

Overview

CYP3A4CYP2C9CYP2D6hERG

OverviewOther

Other InhibitorOther SubstrateOther Inducer

Drug as perpetrator​

Drug as victim

PubMed

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