Chaperone-enzyme replacement therapy product - Amicus/JCR Pharmaceuticals

Drug Profile

Chaperone-enzyme replacement therapy product - Amicus/JCR Pharmaceuticals

Alternative Names: Alpha-galactosidase A/migalastat; Alpha-galactosidase/migalastat; AT-B100; Chaperone-ERT product - Amicus/JCR; JR-051/migalastat; Migalastat/alpha galactosidase; Migalastat/alpha galactosidase A; Migalastat/JR-051

Latest Information Update: 14 Apr 2016

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At a glance

  • Originator Amicus Therapeutics
  • Developer Amicus Therapeutics; JCR Pharmaceuticals
  • Class Enzymes; Pharmacological chaperones; Piperidines; Recombinant proteins; Small molecules
  • Mechanism of Action Alpha-galactosidase stimulants; Protein folding stimulants
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Fabry's disease

Most Recent Events

  • 17 Jul 2012 Preclinical trials in Fabry's disease in USA (Parenteral)
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