Migalastat
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| Trade names | Galafold |
| Synonyms | DDIG, AT1001 |
| AHFS/Drugs.com | UK Drug Information |
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By mouth (capsules) |
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| 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 | 163.17 g·mol−1 |
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Migalastat (INN/USAN; or 1-deoxygalactonojirimycin; trade names Galafold and formerly Amigal) is a drug for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) assigned it orphan drug status in 2004, and the European Committee for Medicinal Products for Human Use (CHMP) followed in 2006. The European Commission approved the drug in May 2016.
Migalastat is used for the long-term treatment of Fabry disease in adults and adolescents aged 16 or older with an amenable mutation of the enzyme alpha-galactosidase A (α-GalA). An "amenable" mutation is one that leads to misfolding of the enzyme, but otherwise would not significantly impair its function.
Based on an in vitro test, Amicus Therapeutics has published a list of 269 amenable and nearly 600 non-amenable mutations. About 35 to 50% of people with Fabry have an amenable mutation.
The most common side effect in clinical trials was headache (in about 10% of people who take it). Less common side effects (between 1 and 10% of people) included unspecific symptoms such as dizziness, fatigue, and nausea, but also depression. Possible rare side effects could not be assessed because of the low number of subjects in the clinical trials in which adverse effects were measured.
When combined with intravenous agalsidase alfa or beta, which are recombinant versions of the enzyme α-GalA, migalastat increases tissue concentrations of functional α-GalA compared to agalsidase given alone. This is an expected and desired effect.
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