Migalastat hydrochloride is a potent and competitive α-galactosidase A (α-Gal A) inhibitor (human α-Gal A,IC50 of 0.04 μM ).
Migalastat hydrochloride toward human lysosomal a-Gal A Both IC50 and Ki values of are 0.04 μM[1].
Oral administration of Migalastat (GR181413A) hydrochloride to transgenic mice reduces elevated lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively, generally equal to or greater than observed for GL-3[3].