Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0MU9L
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| Former ID |
DCL000166
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| Drug Name |
Migalastat
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| Synonyms |
DDIG; D-Galactitol, 1,5-dideoxy-1,5-imino; (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol; 1,5-Dideoxy-1,5-iminogalactitol; 2-(Hydroxymethyl)-3,4,5-piperidinetriol hydrochloride
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| Drug Type |
Small molecular drug
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| Indication | Fabry disease [ICD-11: 5C56.01] | Approved | [1] | |
| Company |
Amicus Therapeutics
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| Structure |
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Download2D MOL |
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| Formula |
C6H13NO4
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| Canonical SMILES |
C1C(C(C(C(N1)CO)O)O)O
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| 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
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| InChIKey |
LXBIFEVIBLOUGU-DPYQTVNSSA-N
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| CAS Number |
CAS 108147-54-2
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| PubChem Compound ID | ||||
| PubChem Substance ID |
796556, 10258334, 11114003, 15066543, 15321768, 33499767, 34884209, 50069796, 76308781, 103329495, 104049440, 104425300, 128553861, 131271591, 131481001, 131481032, 134348144, 135154287, 137263801, 139879276, 144203290, 144221323, 162774201, 172232452, 178483437, 198184004, 198975997, 223733290, 227686116, 249807450, 250135145
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| ChEBI ID |
CHEBI:135923
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Alpha-galactosidase A (GLA) | Target Info | Modulator | [2], [3] |
| KEGG Pathway | Galactose metabolism | |||
| Glycerolipid metabolism | ||||
| Sphingolipid metabolism | ||||
| Glycosphingolipid biosynthesis - globo series | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Sphingolipid Metabolism | |||
| Galactose Metabolism | ||||
| Reactome | Glycosphingolipid metabolism | |||
| WikiPathways | Sphingolipid metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | 2018 FDA drug approvals.Nat Rev Drug Discov. 2019 Feb;18(2):85-89. | |||
| REF 2 | AT-1001: a high-affinity alpha3beta4 nAChR ligand with novel nicotine-suppressive pharmacology. Br J Pharmacol. 2015 Apr;172(7):1834-45. | |||
| REF 3 | The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines. J Inherit Metab Dis. 2009 Jun;32(3):424-40. | |||
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