Migalastat hydrochloride (GR181413A) is an orally available, potent and competitive inhibitor of alpha-galactosidase A. It promotes the transport of alpha-galactosidase A to the lysosome and can be used in the study of Fabry disease.

α-GalA (human):0.04 μM (Ki), α-GalA (human):0.04 μM

Migalastat hydrochloride (GR181413A) demonstrates IC50 and Ki values of 0.04 μM for human lysosomal alpha-Gal A[4].

Fabry disease is an X-linked recessive disorder caused by deficient activity of alpha-galactosidase A (α-Gal A)[2]. In transgenic mice expressing the mutant human α-Gal A (TgM), oral administration of Migalastat hydrochloride (3 mg/kg per day for 4 consecutive weeks) results in a dose- and time-dependent increase in α-Gal A activity in the heart, kidneys, spleen, and liver[2].After a 2-week pretreatment with Migalastat hydrochloride, the half-life for all major issues is less than 1 day[2]. Administration of Migalastat hydrochloride (100 mg/kg per day orally for 28 days) in transgenic mice leads to a reduction of 64%, 59%, and 81% in globotriaosylceramide (Gb3) levels in the kidneys, heart, and skin, respectively[3].