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.
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2D Structure
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Physico-chemical Properties
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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
SMILES / InChI / InChIKey
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Canonical SMILES OCC1NCC(C(C1O)O)O
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
InChI Key LXBIFEVIBLOUGU-DPYQTVNSSA-N
Classification Click here for help
Compound class Synthetic organic
Approved drug? Yes (EMA (2016), FDA (2018))
IUPAC Name Click here for help
(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
International Nonproprietary Names Click here for help
INN number INN
8700 migalastat
Synonyms Click here for help
1-Deoxygalactonojirimycin | Galafold® | GR-181413A | GR181413A
Database Links Click here for help
BindingDB Ligand 50163440
CAS Registry No. 108147-54-2 (source: WHO INN record)
ChEMBL Ligand CHEMBL110458
DrugBank Ligand 5018
DrugCentral Ligand 5110
GtoPdb PubChem SID 381118910
PubChem CID 176077
RCSB PDB Ligand DGJ
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