duvoglustat

Duvoglustat is a small molecule pharmacological chaperone and alpha-glucosidase inhibitor, structurally classified as an iminosugar (alkaloid azasugar). It is being investigated primarily for the treatment of acid α-glucosidase (GAA) deficiency, which causes Pompe disease—a lysosomal storage disorder characterized by progressive accumulation of glycogen in heart and skeletal muscles. Duvoglustat works by stabilizing recombinant human GAA enzyme (alglucosidase alfa), increasing its systemic and tissue exposure when co-administered, thereby enhancing substrate reduction in muscle. In clinical studies, it has shown to increase plasma and muscle GAA activity without significant adverse effects. Duvoglustat also exhibits antihyperglycemic, anti-obesity, and antiviral properties due to its inhibition of carbohydrate digestion enzymes[1][2][3][7].

Other names

duvoglustat hydrochlorideduvoglustat HCl