Drug Profile
Research programme: pharmacological chaperones, enzyme replacements and co-formulations - Amicus Therapeutics
Alternative Names: AT 2221; AT3375; ATB 101/Migalastat; Chaperone monotherapy - Amicus Therapeutics; Chaperone-Enzyme Replacement Therapy - Amicus Therapeutics; Chaperone-ERT combinations - Amicus Therapeutics; Duvoglustate/rhGAA; rhGAA/AT-2220; rhGAA/duvoglustatLatest Information Update: 28 Feb 2023
Price :
$50
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At a glance
- Originator Amicus Therapeutics
- Class Alkaloids; Alpha-glucosidases; Glycoside hydrolases; Imino pyranoses; Pharmacological chaperones; Small molecules
- Mechanism of Action Alpha glucosidase replacements; Glucosylceramidase replacements; Iduronidase replacements; Protein folding stimulants
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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
- Research Fabry's disease
- No development reported Alzheimer's disease; Gaucher's disease; Glycogen storage disease type II; Mucopolysaccharidosis I; Parkinson's disease
Most Recent Events
- 28 Feb 2023 Early research is still ongoing for Fabry's disease in USA (IV) (Amicus Therapeutics pipeline, February 2023)
- 28 Feb 2018 No recent reports of development identified for preclinical development in Glycogen-storage-disease-type-II(Combination therapy) in USA (SC)
- 15 Jan 2018 Early research in Fabry's disease (Combination therapy) in USA (IV)