Eliglustat hemitartrate

Research use only, not for human use.

Eliglustat Hemitartrate, with an IC50 of 24 nM, is a specific, potent, oral active glucocerebral glycosidase inhibitor.Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.A novel inhibitor of glucosylceramide synthase (Genz-112638) and assessed its activity in a murine model of Gaucher disease (D409V/null). Biochemical characterization of Genz-112638 showed good potency (IC(50) approximately 24nM) and specificity against the target enzyme.

Eliglustat Hemitartrate, with an IC50 of 24 nM, is a specific, potent, oral active glucocerebral glycosidase inhibitor.Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.A novel inhibitor of glucosylceramide synthase (Genz-112638) and assessed its activity in a murine model of Gaucher disease (D409V/null). Biochemical characterization of Genz-112638 showed good potency (IC(50) approximately 24nM) and specificity against the target enzyme. Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals. Treatment of older mice that already displayed significant amounts of tissue glucosylceramide (7 months old) resulted in arrest of further accumulation of the substrate and appearance of additional Gaucher cells in affected organs. These data indicate that substrate inhibition therapy with Genz-112638 represents a viable alternate approach to enzyme therapy to treat the visceral pathology in Gaucher disease.

Eliglustat tartrate shows good potency with an IC50 of 24 nM and specificity against the target enzyme.Incubating K562 or B16/F10 cells for 72 h with increasing amounts of Genz-112638 (0.6-1000 nM) results in a dose-dependent reduction of cell surface levels of both GM1 and GM3. The mean IC50 value for inhibiting the cell surface presentation of GM1 in K562 cells was 24 nM (range 14-34 nM) and that for GM3 in B16/F10 cells was 29 nM (range 12-48 nM).

Mice that received drug prior to significant accumulation of substrate (10 weeks of age) showed reduced levels of glucosylceramide and number of Gaucher cells in the spleen, lung and liver when compared to age-matched control animals.

Genz-112638,Eliglustat tartrate

Others

Other Targets

glucocerebroside synthase

Metabolism

Gaucher′s Disease

928659-70-5

959.17

C50H78N4O14

>98% (HPLC)

H2O:50 mg/mL (52.13 mM);DMSO:50 mg/mL (52.13 mM)

CCCCCCCC(N[C@H](CN1CCCC1)[C@@H](C2=CC=C(OCCO3)C3=C2)O)=O.CCCCCCCC(N[C@H](CN4CCCC4)[C@@H](C5=CC=C(OCCO6)C6=C5)O)=O.O=C(O)[C@H](O)[C@@H](O)C(O)=O

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(-20℃)

With Ice Pack

≥ 2 years