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Compound Detail

CHEMBL110458

MIGALASTAT
💊 Approved Drug
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💊 Approved Drug
OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
ChEMBL ↗ Properties Compare Identifier

Basic Information

ChEMBL ID CHEMBL110458
Name MIGALASTAT
Phase Approved
Structure Type MOL
InChI Key LXBIFEVIBLOUGU-DPYQTVNSSA-N
Therapeutic ✓ Yes

Known Names

At-1001, gr-181314a Galafold Migalastat
15
Targets
26
Activities
2
Assay Types
0
PK Parameters
20
Publications
3
Known Names
1
Indications
1
Mechanisms

Molecular Properties

163.2
MW (Da)
-2.97
ALogP
5
HBA
5
HBD
93.0
PSA (Ų)
0.28
QED
0
Ro5 Violations
1
Rot. Bonds

Drug Information

Molecule Type Small molecule
First Approval 2016
Availability Prescription
Chirality Single Enantiomer

Routes of Administration

Oral

Flags

Natural Product First in Class
USAN Stem -stat
USAN Year 2005

Extended Properties

Molecular Formula C6H13NO4
MW 163.17
Aromatic Rings 0
Heavy Atoms 11
NP-Likeness 2.44

Mechanism of Action

Mechanism Action Type Target Direct Efficacy
Alpha-galactosidase A stabiliser STABILISER Alpha-galactosidase A ✓ ✓

Indications

Fabry Disease

ATC Classification

A16AX14
migalastat
Level 1: ALIMENTARY TRACT AND METABOLISM
Level 2: OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS

Activity Summary

IC50 20 meas. best 8.52
Ki 6 meas. best 7.89

Target Activity Profile

Target Type Best pChEMBL Mean pChEMBL Best (nM) # Data
Unchecked
CHEMBL612545
 IC50 8.52 6.5567 3.0 3
Alpha-galactosidase
CHEMBL5217
 IC50 8.52 8.52 3.0 1
Alpha-galactosidase A
CHEMBL2524
 IC50 7.89 7.3975 13.0 4
Alpha-galactosidase
CHEMBL5217
 Ki 7.89 7.89 13.0 1
Sucrase-isomaltase, intestinal
CHEMBL2748
 IC50 6.58 6.58 260.0 1
Maltase-glucoamylase
CHEMBL2074
 IC50 5.82 5.82 1500.0 1
Alpha-galactosidase C
CHEMBL3936
 IC50 5.75 5.75 1800.0 1
Lysosomal alpha-glucosidase
CHEMBL2608
 IC50 5.22 5.22 6000.0 1
Glycogen debranching enzyme
CHEMBL5272
 IC50 5.0 5.0 10000.0 1
Beta-galactosidase
CHEMBL3484
 Ki 4.9 4.9 12500.0 1
Beta-galactosidase
CHEMBL1293264
 Ki 4.89 4.89 13000.0 1
Beta-galactosidase
CHEMBL2680
 IC50 4.62 4.62 24000.0 1
Lactase/phlorizin hydrolase
CHEMBL1075131
 IC50 4.3 4.3 50000.0 1
Lactase/phlorizin hydrolase
CHEMBL3389
 IC50 4.14 4.14 72000.0 1
Beta-galactosidase
CHEMBL2522
 IC50 4.05 4.05 90000.0 1
Beta-glucosidase
CHEMBL3761
 IC50 4.0 4.0 100000.0 1
Unchecked
CHEMBL612545
 Ki 4.0 4.0 100000.0 2

Activity Data Samples

Top measurements by pChEMBL value

Target Type Rel. Value Units pChEMBL Assay PubMed
Alpha-galactosidase IC50 = 3.0 nM 8.52 Inhibition of coffee bean alpha-galactosidase assessed as p-nitrophenol release ... 20457528
Unchecked IC50 = 3.0 nM 8.52 Inhibitory concentration against alpha-galactosidase of coffee bean 15771446
Alpha-galactosidase A IC50 = 13.0 nM 7.89 Inhibition of recombinant human alpha GAL-A using 4-methylumbelliferyl alpha-D-g... 27744182
Alpha-galactosidase Ki = 13.0 nM 7.89 Inhibition of alpha-galactosidase green coffee beans 20610152
Alpha-galactosidase A IC50 = 42.0 nM 7.38 Inhibition of recombinant human alpha GAL-A using 4-methylumbelliferyl alpha-D-g... 27744182
Alpha-galactosidase A IC50 = 70.0 nM 7.16 Inhibitory concentration against human alpha-galactosidase 15771446
Alpha-galactosidase A IC50 = 70.0 nM 7.16 Inhibition of human lysosome alpha-galactosidase assessed as p-nitrophenol relea... 20457528
Unchecked IC50 = 80.0 nM 7.1 Inhibition of alpha-galactosidase in human PBMC using 4-methylumbelliferyl alpha... 27750150
Sucrase-isomaltase, intestinal IC50 = 260.0 nM 6.58 Inhibition of sucrase by HPLC 20000679
Maltase-glucoamylase IC50 = 1500.0 nM 5.82 Inhibition of maltase by HPLC 20000679
Alpha-galactosidase C IC50 = 1800.0 nM 5.75 Inhibitory concentration against alpha-galactosidase of Aspergillus niger 15771446
Lysosomal alpha-glucosidase IC50 = 6000.0 nM 5.22 Inhibition of lysosomal alpha-glucosidase by HPLC 20000679
Glycogen debranching enzyme IC50 = 10000.0 nM 5.0 Inhibition of glycogen glycogen de-branching enzyme by HPLC 20000679
Beta-galactosidase Ki = 12500.0 nM 4.9 Inhibition of Agrobacterium sp. beta-galactosidase 16481162
Beta-galactosidase Ki = 13000.0 nM 4.89 Inhibition of Escherichia coli beta-galactosidase 20610152
Beta-galactosidase IC50 = 24000.0 nM 4.62 Inhibitory concentration against beta-galactosidase of rat 15771446
Lactase/phlorizin hydrolase IC50 = 50000.0 nM 4.3 Inhibition of lactase by HPLC 20000679
Lactase/phlorizin hydrolase IC50 = 72000.0 nM 4.14 Inhibition of rat intestinal lactase assessed as p-nitrophenol release by spectr... 20457528
Beta-galactosidase IC50 = 90000.0 nM 4.05 Inhibitory concentration against human beta-galactosidase 15771446
Unchecked IC50 = 90000.0 nM 4.05 Inhibition of Kluyveromyces lactis beta-galactosidase using o-nitrophenyl-beta-o... 33479653

Literature References

PubMed DOI Target Context # Activities
23363020 10.1021/jm301557k Mus musculus 8
20457528 10.1016/j.bmc.2010.04.048 Unchecked 7
23363020 10.1021/jm301557k Beta-galactosidase 6
37468498 10.1038/s41467-023-40064-9 Thromboxane A2 receptor 4
37468498 10.1038/s41467-023-40064-9 Prostaglandin G/H synthase 1 4
37468498 10.1038/s41467-023-40064-9 Alpha-1A adrenergic receptor 4
37468498 10.1038/s41467-023-40064-9 Muscarinic acetylcholine receptor M1 4
27744182 10.1016/j.ejmech.2016.10.004 Alpha-galactosidase A 4
27474919 10.1016/j.ejmech.2016.07.025 Unchecked 3
15771446 10.1021/jm0495881 Beta-galactosidase 3
37468498 10.1038/s41467-023-40064-9 Acetylcholinesterase 2
15771446 10.1021/jm0495881 Beta-mannosidase 2
37468498 10.1038/s41467-023-40064-9 Alpha-2A adrenergic receptor 2
15771446 10.1021/jm0495881 Alpha-mannosidase 2
37468498 10.1038/s41467-023-40064-9 Sodium-dependent noradrenaline transporter 2
37468498 10.1038/s41467-023-40064-9 Voltage-gated inwardly rectifying potassium channel KCNH2 2
15771446 10.1021/jm0495881 Tissue alpha-L-fucosidase 2
37468498 10.1038/s41467-023-40064-9 Sodium-dependent serotonin transporter 2
37468498 10.1038/s41467-023-40064-9 Sodium-dependent dopamine transporter 2
37468498 10.1038/s41467-023-40064-9 D(1A) dopamine receptor 2

Data from ChEMBL 36 via Core Engine