migalastat   Click here for help

GtoPdb Ligand ID: 10200

Synonyms: 1-Deoxygalactonojirimycin | Galafold® | GR-181413A | GR181413A
Approved drug PDB Ligand
migalastat is an approved drug (EMA (2016), FDA (2018))
Compound class: Synthetic organic
Comment: Migalastat was developed by Amicus Therapeutics as a first-in-class oral monotherapy for the treatment of Fabry disease [2]. Mechanistically it acts as a pharmacological chaperone that stabilises certain mutant forms of α-galactosidase that are found in Fabry disease patients and enhances enzyme activity [1]. It is a stereoisomer of 1-deoxynojirimycin.
Click here for help
2D Structure
Click here for help
Click here for structure editor
Physico-chemical Properties
Click here for help
Hydrogen bond acceptors 5
Hydrogen bond donors 5
Rotatable bonds 1
Topological polar surface area 92.95
Molecular weight 163.08
XLogP -0.93
No. Lipinski's rules broken 0
Click here for help
Isomeric SMILES OC[C@H]1NC[C@@H]([C@H]([C@H]1O)O)O
InChI 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
No information available.
Summary of Clinical Use Click here for help
Migalastat is approved for the long-term treatment of Fabry disease. It is an effective therapeutic alternative to enzyme replacement therapy for suitable patients [2].
Mechanism Of Action and Pharmacodynamic Effects Click here for help
Migalastat binds to and stabilises misfolded/defective forms of α-galactosidase A. This allows the enzyme to be transported into areas of the cell where it can break down cytotoxic sphingolipid globotriaosylceramide (Gb3) that builds up when α-galactosidase A activity is insufficient.
External links Click here for help