| Bioactivity | Migalastat (GR181413A free base) is an orally active and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM for human α-Gal A[1]. |
| Target | IC50: 0.04 μM (human α-Gal A);Ki: 0.04 μM (human α-Gal A) |
| Invitro | Migalastat inhibits human lysosomal a-Gal A with IC50 and Ki values of 0.04 μM[1]. Cell Viability Assay[4] Cell Line: |
| In Vivo | 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]. Animal Model: |
| Name | Migalastat |
| CAS | 108147-54-2 |
| Formula | C6H13NO4 |
| Molar Mass | 163.17 |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | Please store the product under the recommended conditions in the Certificate of Analysis. |
| Reference | [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. |