Lucerastat (NB-DGJ, ACT-434964) is a galactose analogue that is an UDP-glucose ceramide glucosyltransferase (UGCG/GCS) inhibitor (IC50=41.4 uM).
Lucerastat prevents glucosylceramide biosynthesis both in vitro and in mouse models of lysosome storage disorders, without affecting α-glucosidase I and II or β-glucocerebrosidase (IC50>1 mM).
Lucerastat shows potential as substrate reduction therapy for glycolipid storage disorders such as Gaucher disease and Fabry disease.
