Definition

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.