Migalastat HCl | Galactosidase inhibitor/Pharmacological chaperone

CAS:75172-81-5

Catalog Number:10-2632

Migalastat (75172-81-5) is a potent, selective, and orally available inhibitor of α-galactosidase A (IC50= 40 nM).

1 It binds to mutant misfolded α-GalA shifting the folding behavior toward functional active conformation (pharmacological 

chaperone activity) followed by trafficking to the lysosomes.2 Migalastat is active broadly across species from Drosophila3

 to human4. Clinically useful agent for Fabry disease.5

1-Deoxygalactonojirimycin, 75172-81-5, alpha-galactosidase A inhibitor, AT1001, GR181413, Migalastat, 

Migalastat HCl supplier, Migalastat supplier, α-galactosidase A inhibitor

Activity:Galactosidase inhibitor and pharmacological chaperone

Chemical Names:(2R,3S,4R,5S)-2-(Hydroxymethyl)-3,4,5-piperidinetriol HCl

Alternate Name:1-Deoxygalactonojirimycin HCl; DGJ; GR181413; AT1001

Molecular Weight:199.63

Molecular Formula:C6H13O4·HCl

Solubility:Soluble in water (50 mg/ml)

Physical Properties:White solid

Purity:>98% by TLC

NMR: (Conforms)

Storage Temperature:-20°C

Stability:Stable for 2 years from the date of purchase as supplied. Solutions in water may be stored at -20°C for up to 2 months.

Shipping Code:RT

Safety Data Sheet:N/A

References/Citations:

1.Asano et al. (2000), In vitro inhibition and intracellular enhancement of lysosomal α-galactosidase A activity in Fabry 

lymphoblasts by 1-deoxygalactonojirimycin and its derivatives; Eur. J. Biochem. 267 4179

2.Siekierska et al. (2012), α-Galactosidase aggregation is a determinant of pharmacological chaperone efficacy on Fabry 

disease mutants; J. Biol. Chem. 287 28386

3.Braunstein et al. (2020), Misfolding of Lysosomal α-Galactosidase a in a Fly Model and Its Alleviation by the Pharmacological 

Chaperone Migalastat; Int. J. Mol. Sci. 21 7397

4.Benjamin et al. (2009), The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in 

Fabry patient cell lines; J. Inherit. Metab. Dis. 32 424

5.McCafferty and Scott (2019), Migalastat: A Review in Fabry Disease; Drugs 79 543

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