Ibiglustat

Description: Ibiglustat (GZ402671) is a potent and selective Glucosylceramide synthase inhibitor and ceramide glucosyltransferase inhibitor. Ibiglustat blocks the formation of glucosylceramide (GL-1), a key intermediate in the synthesis of GL-3. Ibiglustat is potentially useful for treating Fabry disease. Fabry disease is a rare lysosomal storage disorder, which results in abnormal tissue deposits of a particular fatty substance called globotriaosylceramide (GL-3 or Gb3) throughout the body. Target: Transferase. Smiles: CC(C)(NC(=O)O[C@@H]1CN2CCC1CC2)c1csc(n1)-c1ccc(F)cc1. References: Schiffmann R, et al. Fabry disease. Handb Clin Neurol. 2015,132:231-48.