Drug Profile


Alternative Names: 1-deoxynojirimycin hydrochloride; AT-2220; AT2220-ERT combo therapy; AT2220-IV; Duvoglustat HCl; duvoglustat hydrochloride; duvoglustat-ERT combo therapy; HGT-3510

Latest Information Update: 09 Aug 2015

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At a glance

  • Originator Amicus Therapeutics
  • Class Alkaloids; Imino pyranoses; Pharmacological chaperones; Small molecules
  • Mechanism of Action Alpha-glucosidase stimulants; Protein folding stimulants
  • Orphan Drug Status

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

    Yes - Glycogen storage disease type II
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Glycogen storage disease type II

Most Recent Events

  • 15 Dec 2016 Biomarkers information updated
  • 09 Aug 2015 Clinical development of duvoglustat is ongoing
  • 14 Feb 2013 Amicus Therapeutics plans a repeat-dose trial for Glycogen storage disease type II (IV formulation) in USA
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