Eliglustat

Eliglustat is an specific, potent and orally active glucocerebroside synthase inhibitor with an IC₅₀ of 24 nM. IC50 & Target: IC50: 24 nM (glucocerebroside synthase)^[1] In Vitro: Eliglustat tartrate shows good potency with an IC₅₀ of 24 nM and specificity against the target enzyme^[1].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)^[1]. In Vivo: 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^[1].