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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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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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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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

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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
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
For extended ADME data see the following:

Drugs.com
European Medicines Agency (EMA)