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
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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
1. Asano N, Ishii S, Kizu H, Ikeda K, Yasuda K, Kato A, Martin OR, Fan JQ. (2000)
In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives.
Eur J Biochem, 267 (13): 4179-86. [PMID:10866822]
2. Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T et al.. (2017)
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
J Med Genet, 54 (4): 288-296. [PMID:27834756]