Migalastat

Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity[1].

Product Specifications

CAS Number

[108147-54-2]

Product Name Alternative

GR181413A (free base) ; 1-Deoxygalactonojirimycin

UNSPSC

12352005

Hazard Statement

H315-H319-H320

Target

Glycosidase

Type

Reference compound

Related Pathways

Metabolic Enzyme/Protease

Field of Research

Others

Assay Protocol

https://www.medchemexpress.com/migalastat.html

Solubility

10 mM in DMSO

Smiles

O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O

Molecular Formula

C6H13NO4

Molecular Weight

163.17

Precautions

P264-P280-P302+P352-P305+P351+P338-P362

References & Citations

[1]Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267 (13) :4179-86.|[2]Ishii S, et al. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328 (3) :723-31.|[3]Young-Gqamana B, et al. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. PLoS One. 2013;8 (3) :e57631.|[4]Welford RWD, et al. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct. 27 (19) :3392-3403.