Eliglustat (hemitartrate)

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 alpha-glucosidase I and II, alpha-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.Formal Name: N-[(1R,2R)-2-(2,3-dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(1-pyrrolidinylmethyl)ethyl]-octanamide, 2R,3R-dihydroxybutanedioate (2:1). CAS Number: 928659-70-5. Synonyms: Genz-112638. Molecular Formula: C23H36N2O4 . 1/2C4H6O6. Formula Weight: 479.6. Purity: >98%. Formulation: (Request formulation change), A crystalline solid. Solubility: DMF: 20 mg/ml, DMSO: 20 mg/ml, Ethanol: 20 mg/ml, Ethanol:PBS (pH 7.2) (1:30): 0.03 mg/ml. lambdamax: 286 nm. SMILES: O[C@@H]([C@H](N([H])C(CCCCCCC)=O)CN1CCCC1)C2=CC=C(OCCO3)C3=C2.OC([C@H](O)[C@@H](O)C(O)=O)=O.O[C@@H]([C@H](N([H])C(CCCCCCC)=O)CN4CCCC4)C5=CC=C(OCCO6)C6=C5. InChi Code: InChI=1S/2C23H36N2O4.C4H6O6/c2*1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20,5-1(3(7)8)2(6)4(9)10/h2*10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26),1-2,5-6H,(H,7,8)(H,9,10)/t2*19-,23-,1-,2-/m111/s1. InChi Key: KUBARPMUNHKBIQ-VTHUDJRQSA-N.