Ibiglustat (L-Malic acid)_product_DataBase

Bioactivity

Ibiglustat (Venglustat) L-Malic acid is an orally active, brain-penetrant glucosylceramide synthase (GCS) inhibitor. Ibiglustat L-Malic acid 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].

Target

Glucosylceramide synthase.

Invitro

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

Name

Ibiglustat (L-Malic acid)

CAS

1629063-78-0

Formula

C24H30FN3O7S

Molar Mass

523.57

Appearance

Solid

Transport

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

Reference

[1]. Viel C, et al. Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model. Sci Rep. 2021;11(1):20945. Published 2021 Oct 22. [2]. Peterschmitt MJ, et al. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021;10(1):86-98. [3]. Iva Stojkovska, et al. Molecular mechanisms of α-synuclein and GBA1 in Parkinson’s disease. Cell Tissue Res. 2017. [4]. Itier JM, et al. Effective clearance of GL-3 in a human iPSC-derived cardiomyocyte model of Fabry disease. J Inherit Metab Dis. 2014 Nov;37(6):1013-22.

PeptideDB

Ibiglustat (L-Malic acid)

CAS: 1629063-78-0 F: C24H30FN3O7S W: 523.57