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Migalastat - Amicus Therapeutics

Drug Profile

Migalastat - Amicus Therapeutics

Alternative Names: AT-1001-Amicus-Therapeutics; GALAFOLD; Galafold; GR181413A; GR181413A/AT1001; HGT-3310; Migalastat hydrochloride

Latest Information Update: 06 Mar 2024

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At a glance

  • Originator Amicus Therapeutics
  • Class Imino sugars; Pharmacological chaperones; Piperidines; Small molecules
  • Mechanism of Action Alpha-galactosidase A transport stimulants; Protein folding stabiliser
  • 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

  • Marketed Fabry's disease

Most Recent Events

  • 31 Dec 2023 Migalastat - Amicus Therapeutics receives Orphan Drug status for Fabry's disease in United Kingdom, prior to December 2023
  • 08 Aug 2023 Amicus Therapeutics receives multiple new patent protection for Migalastat in USA
  • 02 Mar 2023 Registered for Fabry's disease (In adolescents, Monotherapy) in United Kingdom (PO)
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