Drug Profile
Migalastat - Amicus Therapeutics
Alternative Names: AT-1001-Amicus-Therapeutics; GALAFOLD; Galafold; GR181413A; GR181413A/AT1001; HGT-3310; Migalastat hydrochlorideLatest Information Update: 06 Mar 2024
Price :
$50
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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
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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
- 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)