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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| 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,[5] and the European Commission followed in 2006.[6] 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.[7][8][9]
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[10]
- ^ "Galafold (Amicus Therapeutics Pty Ltd)". tga.gov.au. Retrieved 29 March 2023.
- ^ "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.
- ^ "Prescription medicines and biologicals: TGA annual summary 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 31 March 2024.
- ^ "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.
- ^ "Migalastat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Archived from the original on October 31, 2021. Retrieved 16 September 2020.
- ^ "EU/3/06/368". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
- ^ Cite error: The named reference
Newswirewas invoked but never defined (see the help page). - ^ Cite error: The named reference
EPARwas invoked but never defined (see the help page). - ^ "Galafold EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
- ^ New Drug Therapy Approvals 2018. U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original (PDF) on August 26, 2019. Retrieved 16 September 2020.