Eliglustat CAS: 491833-29-5

Eliglustat is an specific, potent and orally active glucocerebroside synthase inhibitor with an IC50 of 24 nM.
Eliglustat is a carboxamide obtained by formal condensation of the carboxy group of octanoic acid with the primary amino group of (1R,2R)-2-amino-1-(2,3-dihydro-1,4-benzodioxin-6-yl)-3-(pyrrolidin-1-yl)propan-1-ol. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease. It has a role as an EC 2.4.1.80 (ceramide glucosyltransferase) inhibitor. It is a benzodioxine, a N-alkylpyrrolidine, a secondary alcohol and a carboxamide. It is a conjugate base of an eliglustat(1+).|Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease. Patients selected for treatment with Eliglustat undergo an FDA approved genotype test to establish if they are CYP2D6 EM (extensive metabolizers), IM (intermediate metabolizers), or PM (poor metabolizers), as the results of this test dictate the dosage of Eliglustat recommended. There are no recommended dosing guidelines for CYP2D6 ultra-rapid or indeterminate metabolizers. Eliglustat was approved for use by the FDA in August 2014.|Eliglustat is a Glucosylceramide Synthase Inhibitor. The mechanism of action of eliglustat is as a Glucosylceramide Synthase Inhibitor, and P-Glycoprotein Inhibitor, and Cytochrome P450 2D6 Inhibitor.|Eliglustat is an oral inhibitor of glucosylceramide synthase which is used in the therapy of type 1 Gaucher disease. Clinical experience with eliglustat is limited, but it not been linked to serum enzyme elevations during therapy or to instances of clinically apparent acute liver injury.|Eliglustat is a ceramide analog and an orally bioavailable inhibitor of glucosylceramide synthase (GCS; ceramide glucosyltransferase), that may be used to decrease the production of glycosphingolipids (GSLs) including glucosylceramide, also known as glucocerebroside. Upon oral administration, eliglustat inhibits GCS, which catalyzes the initial step in the synthesis of glucosylceramide and other GSLs. This decreases the production of glucosylceramide and other GSLs, which have important roles in various diseases and cellular processes, including immune processes and functions. Eliglustat may be used in substrate reduction therapy in diseases in which the enzyme glucocerebrosidase (GCase), responsible for the breakdown of glucocerebroside, is deficient.