Compound Detail
CHEMBL1029
MIGLUSTAT 💊 Approved Drug
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💊 Approved Drug
Basic Information
| ChEMBL ID | CHEMBL1029 |
| Name | MIGLUSTAT |
| Phase | Approved |
| Structure Type | MOL |
| InChI Key | UQRORFVVSGFNRO-UTINFBMNSA-N |
| Therapeutic | ✓ Yes |
| Active Moiety | CHEMBL4297283 |
20
Targets
71
Activities
3
Assay Types
0
PK Parameters
20
Publications
10
Known Names
13
Indications
1
Mechanisms
Molecular Properties
219.3
MW (Da)
-1.45
ALogP
5
HBA
4
HBD
84.2
PSA (Ų)
0.46
QED
0
Ro5 Violations
4
Rot. Bonds
Drug Information
| Molecule Type | Small molecule |
| First Approval | 2002 |
| Availability | Prescription |
| Chirality | Single Enantiomer |
Mechanism of Action
| Mechanism | Action Type | Target | Direct | Efficacy |
|---|---|---|---|---|
| Ceramide glucosyltransferase inhibitor | INHIBITOR | Ceramide glucosyltransferase | ✓ | ✓ |
Indications
Gaucher Disease Gaucher Disease Glycogen Storage Disease Type II Niemann-Pick Diseases Gangliosidoses, GM2 Glycogen Storage Disease Niemann-Pick Disease, Type C Sandhoff Disease Cystic Fibrosis HIV Infections Spastic Paraplegia, Hereditary Diarrhea Neuronal Ceroid-Lipofuscinoses
ATC Classification
A16AX06
miglustat
Level 1: ALIMENTARY TRACT AND METABOLISM
Level 2: OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
Activity Summary
IC50 64 meas. best 7.85
EC50 4 meas. best 4.93
Ki 3 meas. best 6.7
Target Activity Profile
| Target | Type | Best pChEMBL | Mean pChEMBL | Best (nM) | # Data |
|---|---|---|---|---|---|
| Beta-glucosidase CHEMBL3761 | IC50 | 7.85 | 6.7957 | 14.0 | 7 |
| Lysosomal alpha-glucosidase CHEMBL2608 | IC50 | 7.0 | 7.0 | 100.0 | 2 |
| Non-lysosomal glucosylceramidase CHEMBL5614 | IC50 | 6.85 | 6.85 | 140.0 | 1 |
| Lysosomal alpha-glucosidase CHEMBL2608 | Ki | 6.7 | 6.7 | 200.0 | 1 |
| Alpha-glucosidase CHEMBL5280 | IC50 | 6.38 | 6.38 | 420.0 | 1 |
| Sucrase-isomaltase, intestinal CHEMBL2748 | IC50 | 6.3 | 6.3 | 500.0 | 1 |
| Sucrase-isomaltase, intestinal CHEMBL3114 | IC50 | 6.24 | 5.3014 | 580.0 | 7 |
| Sucrase-isomaltase CHEMBL1667666 | IC50 | 6.22 | 6.02 | 600.0 | 2 |
| Unchecked CHEMBL612545 | IC50 | 6.0 | 5.14 | 1000.0 | 6 |
| CuFi-1 CHEMBL4513124 | IC50 | 5.7 | 5.7 | 2000.0 | 1 |
| Maltase-glucoamylase CHEMBL2074 | IC50 | 5.68 | 5.365 | 2100.0 | 2 |
| Acidic alpha-glucosidase CHEMBL3513 | IC50 | 5.68 | 5.48 | 2100.0 | 2 |
| Unchecked CHEMBL612545 | Ki | 5.43 | 5.43 | 3700.0 | 1 |
| Lysosomal alpha-glucosidase CHEMBL1667668 | IC50 | 5.05 | 5.05 | 9000.0 | 1 |
| Glycogen debranching enzyme CHEMBL5272 | IC50 | 5.0 | 5.0 | 10000.0 | 1 |
| Lysosomal alpha-glucosidase CHEMBL1667668 | EC50 | 4.93 | 4.93 | 11800.0 | 1 |
| Trehalase CHEMBL3248 | IC50 | 4.89 | 4.655 | 13000.0 | 2 |
| Ceramide glucosyltransferase CHEMBL2063 | IC50 | 4.7 | 4.584 | 20000.0 | 5 |
| Ceramide glucosyltransferase CHEMBL6013 | IC50 | 4.64 | 4.425 | 22900.0 | 4 |
| Uncharacterized protein CHEMBL2176863 | IC50 | 4.44 | 4.44 | 36000.0 | 1 |
| SARS-CoV-2 CHEMBL4303835 | IC50 | 4.38 | 4.38 | 42200.0 | 1 |
| Unchecked CHEMBL612545 | EC50 | 4.25 | 4.25 | 56400.0 | 1 |
| Bovine viral diarrhea virus 1 CHEMBL612571 | EC50 | 4.0 | 4.0 | 100000.0 | 1 |
| Dengue virus CHEMBL613757 | EC50 | 4.0 | 4.0 | 100000.0 | 1 |
Activity Data Samples
Top measurements by pChEMBL value
Literature References
Data from ChEMBL 36 via Core Engine