 | |
| Clinical data | |
|---|---|
| Pronunciation | mi GAL a stat |
| Trade names | Galafold |
| Other names | DDIG, AT1001, 1-deoxygalactonojirimycin |
| AHFS/Drugs.com | Monograph |
| License data | |
| Pregnancy category |
|
| Routes of administration | By mouth (capsules) |
| Drug class | Chaperone[1] |
| Legal status | |
| Legal status | |
| Pharmacokinetic data | |
| Bioavailability | 75% |
| Protein binding | None |
| Metabolites | O-glucuronides (<15%) |
| Elimination half-life | 3–5 hours (single dose) |
| Excretion | Urine (77%), feces (20%) |
| Identifiers | |
| |
| Chemical and physical data | |
| Formula | C6H13NO4 |
| Molar mass | 163.173 g·mol−1 |
| 3D model (JSmol) | |
| |
| |
Migalastat, sold under the brand name Galafold, is a medication used to treat Fabry disease.[2] It is only used for certain types of the disease.[2] It is taken by mouth.[2] It is used in those who are at least 12 years old.[3]
Common side effects include headache, runny nose, fever, nausea, and urinary tract infection.[3][2] Safety in pregnancy is unclear.[4] It works by attaching to certain forms of the enzyme alpha-galactosidase A and stabilizing it.[3]
Migalastat was approved for medical use in Europe in 2016 and the United States in 2018.[3][2] In the United States it costs about 315,000 USD per year as of 2020.[5] In the United Kingdom this amount costs the NHS about £121,000.[1]
References edit
- ^ a b BNF 81: March-September 2021. BMJ Group and the Pharmaceutical Press. 2021. p. 1112. ISBN 978-0857114105.
- ^ a b c d e f g "Migalastat Monograph for Professionals". Drugs.com. Archived from the original on 19 January 2021. Retrieved 18 November 2021.
- ^ a b c d e "Galafold EPAR". European Medicines Agency (EMA). Archived from the original on 20 September 2020. Retrieved 16 September 2020.
- ^ "Migalastat (Galafold) Use During Pregnancy". Drugs.com. Archived from the original on 28 November 2020. Retrieved 18 November 2021.
- ^ "How much does Galafold cost?". Drugs.com. Archived from the original on 19 April 2021. Retrieved 18 November 2021.