Eliglustat

Generic Name
Eliglustat
Brand Names
Cerdelga
Drug Type
Small Molecule
Chemical Formula
C23H36N2O4
CAS Number
491833-29-5
Unique Ingredient Identifier
DR40J4WA67
Background

Eliglustat is a glucosylceramide synthase inhibitor used for the long-term treatment of type 1 Gaucher disease. Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide into glucose and ceramide. In patients with Gaucher disease, the accumulation of glucosylceramide leads to th...

Indication

Eliglustat is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease in adult patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. CYP2D6 ultra-rapid metabolizers may not achieve adequate eliglustat concentrations to...

Associated Conditions
Gaucher Disease, Type 1
Associated Therapies
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