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Miglustat - Actelion Pharmaceuticals

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Drug Profile

Miglustat - Actelion Pharmaceuticals

Alternative Names: AT2221; Brazaves; NB-DNJ; OGT 918; OXAIDS; SC 48334; Zavesca

Latest Information Update: 17 Oct 2025

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At a glance

  • Originator Oxford Glycobiology Institute
  • Developer Actelion Pharmaceuticals; National Taiwan University; Teva Pharmaceutical Industries; United Therapeutics Corporation
  • Class Alcohols; Alkaloids; Alkanes; Imino pyranoses; Piperidines; Small molecules
  • Mechanism of Action Alpha-glucosidase inhibitors; Glucosylceramide synthase inhibitors
  • Orphan Drug Status

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

    Yes - Gaucher's disease type I; Niemann-Pick diseases; Niemann-Pick disease type C
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Gaucher's disease type I; Niemann-Pick disease type C
  • Phase II Cystic fibrosis; Gaucher's disease type III
  • No development reported Hepatitis C
  • Discontinued HIV infections; Tay-Sachs disease

Most Recent Events

  • 13 Oct 2025 ADVANZ PHARMA acquires global rights to miglustat (Zavesca®) from Actelion Pharmaceuticals
  • 10 Apr 2019 Actelion Pharmaceuticals plans a phase IV trial for Niemann-Pick diseases (In children, In adolescents, In adults, In the elderly) in China (PO, Capsule) in July 2019 (NCT03910621)
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Hepatitis-C in USA (PO)

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