Drug Profile


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

Latest Information Update: 10 Sep 2017

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At a glance

  • Originator Oxford Glycobiology Institute
  • Developer Actelion Pharmaceuticals; National Taiwan University; Teva Pharmaceutical Industries; United Therapeutics Corporation
  • Class Alkaloids; Imino pyranoses; 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
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Gaucher's disease type I; Niemann-Pick diseases
  • Phase II Cystic fibrosis; Gaucher's disease type III
  • Preclinical Hepatitis C
  • Discontinued HIV infections; Tay-Sachs disease

Most Recent Events

  • 16 Jun 2017 Actelion Pharmaceuticals has been acquired by Janssen Pharmaceuticals
  • 01 Jul 2015 Phase-II clinical trials in Gaucher's disease type III (Combination therapy) in Taiwan (PO) (NCT02520934)
  • 10 Apr 2015 Miglustat is still awaiting approval for Niemann-Pick disease in USA
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