Afegostat

Afegostat (also known as isofagomine) is a small molecule iminosugar that was developed as an experimental oral therapy for certain forms of Gaucher's disease. It acts as a pharmacological chaperone by binding selectively to misfolded β-glucocerebrosidase (GBA), particularly the N370S mutant enzyme found in Gaucher's disease patients. By stabilizing and restoring the correct conformation of this lysosomal enzyme, afegostat enhances its activity and promotes proper metabolism of glucocerebroside. The drug was developed by Amicus Therapeutics and Shire but its development was terminated after a failed clinical trial in 2009[1][3][4][5].

Brand names

Plicera

Other names

afegostatisofagomineiso-fagomineD-isofagomine5-hydroxymethyl-3,4-piperidinediol