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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.

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Correspondence to Raewyn M. Poole.

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This profile has been extracted and modified from the Adis R&D Insight drug pipeline database. Adis R&D Insight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch.

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Poole, R.M. Eliglustat: First Global Approval. Drugs 74, 1829–1836 (2014). https://doi.org/10.1007/s40265-014-0296-3

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Keywords

  • Enzyme Replacement Therapy
  • Liver Volume
  • Lumbar Spine Bone Mineral Density
  • Dabigatran Etexilate
  • Miglustat