Eliglustat hemitartrate 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.
in vitro
Eliglustat tartrate (Eliglustat hemitartrate) shows good potency with an IC50 of 24 nM and specificity against the target enzyme. Incubating K562 or B16/F10 cells for 72 h with increasing amounts of Genz-112638 (0.6-1000 nM) results in a dose-dependent reduction of both GM1 and GM3 cell surface levels. The mean IC50 value for inhibiting the cell surface presentation of GM1 in K562 cells was 24 nM (range 14-34 nM), and that for GM3 in B16/F10 cells was 29 nM (range 12-48 nM).
Eliglustat hemitartrate Upstream-Materialien And Downstream Produkte
Upstream-Materialien
Downstream Produkte