Migalastat

Not to be confused with miglustat.
Migalastat
Clinical data
Pronunciationmi GAL a stat
Trade namesGalafold
Other namesDDIG, AT1001, 1-deoxygalactonojirimycin
AHFS/Drugs.comMonograph
Pregnancy
category
  • AU: B3
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Pharmacokinetic data
Bioavailability75%
Protein bindingNone
MetabolitesO-glucuronides (<15%)
Elimination half-life3–5 hours (single dose)
ExcretionUrine (77%), feces (20%)
Identifiers
  • (2R,3S,4R,5S)-2-(Hydroxymethyl)-3,4,5-piperidinetriol
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC6H13NO4
Molar mass163.173 g·mol−1
3D model (JSmol)
  • C1[C@@H]([C@H]([C@H]([C@H](N1)CO)O)O)O
  • InChI=1S/C6H13NO4/c8-2-3-5(10)6(11)4(9)1-7-3/h3-11H,1-2H2/t3-,4+,5+,6-/m1/s1
  • Key:LXBIFEVIBLOUGU-DPYQTVNSSA-N

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]

  1. ^ "Galafold (Amicus Therapeutics Pty Ltd)". tga.gov.au. Retrieved 29 March 2023.
  2. ^ "Prescription medicines: registration of new chemical entities in Australia, 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 9 April 2023.
  3. ^ "Prescription medicines and biologicals: TGA annual summary 2017". Therapeutic Goods Administration (TGA). 21 June 2022. Retrieved 31 March 2024.
  4. ^ "Genetic disorders". Health Canada. 9 May 2018. Retrieved 13 April 2024.
  5. ^ "Migalastat Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Retrieved 16 September 2020.
  6. ^ "EU/3/06/368". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
  7. ^ Cite error: The named reference Newswire was invoked but never defined (see the help page).
  8. ^ Cite error: The named reference EPAR was invoked but never defined (see the help page).
  9. ^ "Galafold EPAR". European Medicines Agency (EMA). 17 September 2018. Retrieved 16 September 2020.
  10. ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.