Cerdelga (eliglustat) is a prescription medication used for the long-term treatment of adults with Gaucher disease type 1, a rare genetic disorder. Gaucher disease type 1 is caused by a deficiency of the enzyme glucocerebrosidase, which leads to the accumulation of certain fatty substances in various organs, particularly the liver, spleen, and bone marrow. This accumulation can cause a range of symptoms, including enlargement of the liver and spleen, anemia, excessive bleeding and bruising, bone disease, and fatigue. Cerdelga works by inhibiting the synthesis of glucosylceramide, a fatty substance that accumulates in the cells of people with Gaucher disease. By reducing the production of glucosylceramide, Cerdelga helps to manage the symptoms and prevent the progression of the disease. It is an oral medication, making it a convenient option for many patients.