Eliglustat: first global approval.

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-naïve 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.

• Authors: Raewyn M Poole
• Journal: Drugs (Drugs) Vol. 74 Issue 15 Pg. 1829-36 (Oct 2014) ISSN: 1179-1950 [Electronic] New Zealand
• PMID: 25239269 (Publication Type: Journal Article, Review)

Chemical References

• Enzyme Inhibitors
• Pyrrolidines
• eliglustat
• Glucosyltransferases
• ceramide glucosyltransferase

Topics

• Drug Approval
• Enzyme Inhibitors (chemistry, pharmacology, therapeutic use)
• Gaucher Disease (drug therapy)
• Glucosyltransferases (antagonists & inhibitors, metabolism)
• Humans
• Pyrrolidines (chemistry, pharmacology, therapeutic use)