migalastat

Migalastat is an oral small molecule pharmacological chaperone used to treat adults with Fabry disease who have amenable variants in the galactosidase alpha (GLA) gene. It works by selectively and reversibly binding to the active site of the misfolded alpha-galactosidase A (alpha-Gal A) enzyme, stabilizing it and facilitating its proper trafficking from the endoplasmic reticulum to lysosomes. In lysosomes, at acidic pH and high substrate concentration, migalastat dissociates from alpha-Gal A, allowing the enzyme to degrade accumulated glycosphingolipids such as globotriaosylceramide (GL-3) and globotriaosylsphingosine (lyso-Gb3). This mechanism helps restore enzymatic activity in patients with certain GLA mutations that produce unstable but potentially functional forms of alpha-Gal A. Migalastat is developed by Amicus Therapeutics and marketed under the brand name Galafold[2][3][5][6].

Brand names

Galafold

Other names

migalastat hydrochloride