[CAS NO. 1401090-53-6] Ibiglustat

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PRODUCTS SPECIFICATIONS [1401090-53-6]

Catalog

HY-16743

Brand

MCE

CAS

1401090-53-6

DESCRIPTION [1401090-53-6]

Overview

MDL	MFCD28502073
Molecular Weight	389.49
Molecular Formula	C20H24FN3O2S
SMILES	O=C(O[C@@H]1CN2CCC1CC2)NC(C)(C3=CSC(C4=CC=C(F)C=C4)=N3)C

For research use only. We do not sell to patients.

1 Publications Citing Use of MCE

[1]FASEB J. 2020 Dec;34(12):15922-15945.

Summary

Ibiglustat (Venglustat) is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat can be used for the research of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease ^[1] ^[2] .

IC50 & Target

Glucosylceramide synthase ^[1] .

In Vitro

Ibiglustat (SAR402671) (1 μM, 15 days; Fabry disease (FD) cells) is close to the physiological level in untreated WT cells in GL-3 levels, suggesting that Ibiglustat can prevent additional GL-3 accumulation and could serve to ameliorate the abundant levels of this sphingolipid in FD cardiomyocytes ^[4]

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Clinical Trial

NCT Number	Sponsor	Condition	Start Date	Phase
NCT05238714	Genzyme, a Sanofi Company\|Sanofi	Disorders of Sphingolipid Metabolism (Healthy Volunteers)	May 26, 2020	Phase 1
NCT03523728	Genzyme, a Sanofi Company\|Sanofi	Polycystic Kidney, Autosomal Dominant	October 4, 2018	Phase 2\|Phase 3
NCT02906020	Genzyme, a Sanofi Company\|Sanofi	Parkinson´s Disease	December 15, 2016	Phase 2
NCT04705051	Sanofi	Congenital Cystic Kidney Disease	February 9, 2021	Phase 3
NCT05222906	Genzyme, a Sanofi Company\|Sanofi	Gaucher´s Disease Type III	April 18, 2022	Phase 3
NCT05206773	Genzyme, a Sanofi Company\|Sanofi	Fabry Disease	March 11, 2022	Phase 3
NCT05280548	Genzyme, a Sanofi Company\|Sanofi	Fabry´s Disease	May 3, 2022	Phase 3
NCT03687554	Genzyme, a Sanofi Company\|Sanofi	Healthy Volunteers\|Polycystic Kidney, Autosomal Dominant	October 5, 2018	Phase 1
NCT02843035	Genzyme, a Sanofi Company\|Sanofi	Gaucher Disease Type 1\|Gaucher Disease Type 3	January 4, 2017	Phase 2
NCT04221451	Genzyme, a Sanofi Company\|Sanofi	Tay-Sachs Disease Sandhoff Disease	June 29, 2020	Phase 3

Appearance

Solid

Shipping

Room temperature in continental US; may vary elsewhere.

Storage

Powder	-20°C	3 years
	4°C	2 years
In solvent	-80°C	6 months
	-20°C	1 month

Solvent & Solubility

In Vitro:

DMSO : 50 mg/mL ( 128.37 mM ; Need ultrasonic)

Preparing
Stock Solutions

Concentration Solvent Mass	1 mg	5 mg	10 mg

1 mM	2.5675 mL	12.8373 mL	25.6746 mL
5 mM	0.5135 mL	2.5675 mL	5.1349 mL
10 mM	0.2567 mL	1.2837 mL	2.5675 mL

* Please refer to the solubility information to select the appropriate solvent.

In Vivo:

1.

Add each solvent one by one: 10% DMSO >> 40% PEG300 >> 5% Tween-80 >> 45% saline

Solubility: ≥ 2.5 mg/mL (6.42 mM); Clear solution
2.

Add each solvent one by one: 10% DMSO >> 90% (20% SBE-β-CD in saline)

Solubility: ≥ 2.5 mg/mL (6.42 mM); Clear solution
3.

Add each solvent one by one: 10% DMSO >> 90% corn oil

Solubility: ≥ 2.5 mg/mL (6.42 mM); Clear solution

* All of the co-solvents are available by MCE.