Lucerastat - Idorsia Pharmaceuticals

Drug Profile

Lucerastat - Idorsia Pharmaceuticals

Alternative Names: ACT-434964; OGT-923

Latest Information Update: 28 Mar 2018

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At a glance

  • Originator Actelion Pharmaceuticals
  • Developer Actelion Pharmaceuticals; Idorsia Pharmaceuticals
  • Class Antihyperlipidaemics; Piperidines; Small molecules
  • Mechanism of Action Glucosylceramide synthase inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Fabry's disease
  • New Molecular Entity Yes

Highest Development Phases

  • No development reported Fabry's disease; Lipid metabolism disorders

Most Recent Events

  • 28 Mar 2018 No recent reports of development identified for phase-I development in Fabry's-disease in Germany (PO, Capsule)
  • 06 Feb 2018 Actelion plans a pivotal phase III trial for Fabry's disease in the first half of 2018 (NCT03425539)
  • 06 Feb 2018 Lucerastat receives Orphan Drug status for Fabry's disease in European Union before February 2018
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