Eliglustat-(hemi-tartrate)

Eliglustat is an inhibitor of glucosylceramide synthase (IC50 = 40 nM for inhibition of glucosylceramide production in K562 cells). It is selective for glucosylceramide synthase over α-glucosidase I and II, α-1,6-glucosidase, lysosomal glucocerebrosidase, non-lysosomal glucosylceramidase, sucrase, and maltase (IC50s = >10 µM for all). It decreases cell surface levels of the gangliosides GM1 and GM3 in K562 and B16/F10 cells with IC50 values of 24 and 29 nM, respectively. Eliglustat (150 mg/kg per day) decreases glucosylceramide levels in the liver and lungs of D409V/null mice, a model of Gaucher...