The Medical Letter on Drugs and Therapeutics
Eliglustat (Cerdelga) - An Oral Drug for Gaucher Disease (online only)
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The FDA has approved eliglustat (Cerdelga – Genzyme), an oral glucosylceramide synthase inhibitor, for treatment of adults with type 1 Gaucher disease. Eliglustat is metabolized primarily by CYP2D6. Because patients who are CYP2D6 ultra-rapid metabolizers may not achieve therapeutic concentrations and a specific dosage cannot be recommended for indeterminate metabolizers, the FDA has approved the drug only for patients who are extensive, intermediate, or poor metabolizers of CYP2D6.

STANDARD TREATMENT — Patients with Gaucher disease have a genetic deficiency of the lysosomal enzyme glucocerebrosidase that leads to accumulation of glucosylceramide in the lysosomes of reticuloendothelial cells, primarily in the liver, spleen, and bone marrow. The most common form of the disease (type 1) is characterized by hepatosplenomegaly, hematologic disorders, and bone involvement. The standard treatment for Gaucher ... more      

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Title: Eliglustat (Cerdelga) - An Oral Drug for Gaucher Disease (online only)
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