Eliglustat: first global approval.

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Poole RM

Eliglustat: first global approval.

Drugs. 2014 Oct;74(15):1829-36. doi: 10.1007/s40265-014-0296-3.

PubMed ID

25239269 [ View in PubMed

]

Abstract

Eliglustat [Cerdelga (US, EU)], a small-molecule oral glucosylceramide analogue that inhibits the enzyme glucosylceramide synthase has been developed by Genzyme Corporation (a subsidiary of Sanofi) for the treatment of Gaucher disease type 1 in adults. Inhibition of this enzyme reduces the accumulation of the lipid glucosylceramide in the liver, spleen, bone marrow and other organs. Eliglustat received its first global approval in this indication in the US, for use in treatment-naive and treatment-experienced adult patients. It is also under regulatory review in the EU and Japan. This article summarizes the milestones in the development of eliglustat leading to this first approval for Gaucher disease type 1.

DrugBank Data that Cites this Article

Drugs

Eliglustat

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Eliglustat	Ceramide glucosyltransferase	Protein	Humans	Yes	Inhibitor	Details

Drug Enzymes

Drug	Enzyme	Kind	Organism	Pharmacological Action	Actions
Eliglustat	Cytochrome P450 2D6	Protein	Humans	No	Substrate Inhibitor	Details
Eliglustat	Cytochrome P450 3A4	Protein	Humans	No	Substrate	Details

Drug Transporters

Drug	Transporter	Kind	Organism	Pharmacological Action	Actions
Eliglustat	P-glycoprotein 1	Protein	Humans	No	Substrate Inhibitor	Details