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Lucerastat - Idorsia Pharmaceuticals

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Drug Profile

Lucerastat - Idorsia Pharmaceuticals

Alternative Names: ACT 434964; OGT 923

Latest Information Update: 19 Dec 2024

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At a glance

  • Originator Actelion Pharmaceuticals
  • Developer Idorsia Pharmaceuticals
  • Class 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

Most Recent Events

  • 19 Dec 2024 No development reported - Phase-III for Fabry's disease in Japan (PO)
  • 19 Dec 2024 No development reported - Phase-III for Fabry's disease in USA, Austria, Canada, Czech Republic, France, Germany, Netherlands, Poland, United Kingdom, Australia, Ireland, Spain, Norway, Italy, Belgium, Switzerland (PO)
  • 13 Dec 2021 Idorsia Pharmaceuticals plans a meeting with health authorities to define the regulatory pathway for lucerastat in Fabry disease in the first half of 2022

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