Элиглустат (тартрат)

Eliglustat tartrate, developed and marketed by Genzyme Corporation (a subsidiary of Sanofi), was approved by the US FDA in August 2014 for the treatment of nonneuropathic (type 1) Gaucher disease (GD1) in both treatment-naive and treatment-experienced adult patients. It is the first oral treatment to be approved for first-line use in patients with Gaucher disease type 1, which is a rare lysosomal storage disease characterized by accumulation of lipid glucosylceramide (GL-1) due to insufficient production of the enzyme glucosylceramidase. Clinical complications include hepatosplenomegaly, anemia, thrombocytopenia, and bone involvement. Eliglustat is a specific inhibitor of glucosylceramide synthase with an IC50 of 10 ng/mL and acts as substrate reduction therapy for GD1; it has demonstrated non-inferiority to enzyme replacement therapy, which is the current standard of care, in Phase III trials.

Eliglustat Tartrate functions as an oral agent for treatment of Gaucher disease type 1, and lysosomal storage disorders in human. It also inhibits UDP-glucosylceramide synthase in mice thereby preventing Gaucher disease-associated with B-cell malignancy.

ChEBI: A tartrate that is the hemitartrate salt of eliglustat. A ceramide glucosyltransferase inhibitor used (as its tartrate salt) for treatment of Gaucher's disease.