Eliglustat (Tartrate) Chemische Eigenschaften,Einsatz,Produktion Methoden
Beschreibung
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.
Verwenden
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.
Definition
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.
Eliglustat (Tartrate) Upstream-Materialien And Downstream Produkte
Upstream-Materialien
Downstream Produkte
