Migalastat
| Clinical data | |
|---|---|
| Pronunciation | mi GAL a stat |
| Trade names | Galafold |
| Other names | DDIG, AT1001, 1-deoxygalactonojirimycin |
| AHFS/Drugs.com | Monograph |
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| Routes of administration | By mouth |
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| Pharmacokinetic data | |
| Bioavailability | 75% |
| Protein binding | None |
| Metabolites | O-glucuronides (<15%) |
| Elimination half-life | 3–5 hours (single dose) |
| Excretion | Urine (77%), feces (20%) |
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| Chemical and physical data | |
| Formula | C6H13NO4 |
| Molar mass | 163.173 g·mol−1 |
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Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016.
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.