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Eliglustat is an inhibitor of glucosylceramide synthase (IC50 = 40 nM for inhibition of glucosylceramide production in K562 cells).1 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 disease. Formulations containing eliglustat have been used in the treatment of type 1 Gaucher disease.
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1. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease. Mol. Genet. Metab. 91(3), 259-267 (2007).