Migalastat  (Synonyms: GR181413A free base)

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].

IC50: 0.04 μM (human α-Gal A)^[1];
Ki: 0.04 μM (human α-Gal A)^[1]

Migalastat inhibits human lysosomal a-Gal A with IC₅₀ and K_i values of 0.04 μM^[1].

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

Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A^[2].
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM)^[2].
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment^[2].
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively^[3].

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

163.17

C₆H₁₃NO₄

108147-54-2

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

Room temperature in continental US; may vary elsewhere.

Please store the product under the recommended conditions in the Certificate of Analysis.

• [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.  [Content Brief]

  [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.  [Content Brief]

  [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.  [Content Brief]

  [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.  [Content Brief]