Molecule Details
InChIKeyLXBIFEVIBLOUGU-DPYQTVNSSA-N
Compound NameMigalastat
Canonical SMILESOC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
Clinical Status Data-mined Candidate
Targets (Human+Pathogen)2
Pfam Stratification Cross-Family
Avg pChEMBL6.92
SourceChEMBL;BindingDB
2D Structure
2D structure
Activity Profile
DrugBank Annotations
DrugBank ID DB05018
Drug NameMigalastat
CAS Number108147-54-2
Groups approved investigational
ATC Codes A16AX14
DescriptionFabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A). This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds...

Categories: Alimentary Tract and Metabolism Alkaloids Alpha-Galactosidase A (alpha-Gal A) Pharmacological Chaperones Carbohydrates Drugs that are Mainly Renally Excreted Imino Pyranoses Imino Sugars Piperidines Various Alimentary Tract and Metabolism Products
Cross-references: BindingDB: 50163440 ChEBI: 135923 CHEMBL110458 ChemSpider: 153388 Drugs Product Database (DPD): 22894 PDB: DGJ PubChem:176077 PubChem:347827704 RxCUI: 2054252 Wikipedia: Migalastat ZINC: ZINC000001636704
Target Activities (2)
Target Gene Organism Category Pfam pChEMBL Type Source
P06280 GLA Homo sapiens Human PF16499 PF17450 7.3 IC50 ChEMBL;BindingDB
P14410 SI Homo sapiens Human PF13802 PF01055 PF21365 PF00088 6.6 IC50 ChEMBL;BindingDB
DrugBank Target Actions (4)
Target Gene Target Name Action Type
P22309 UGT1A1 UDP-glucuronosyltransferases (UGTs) substrate enzymes
P06280 GLA Alpha-galactosidase A stabilization targets
P13866 SLC5A1 Sodium/glucose cotransporter 1 inhibitor transporters
P13866 SLC5A1 Sodium/glucose cotransporter 1 substrate transporters