Drug Information

Drug General Information
Drug ID
D0MU9L
Former ID
DCL000166
Drug Name
Migalastat
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
Drug Type
Small molecular drug
Indication Fabry's disease [ICD9: 272.7; ICD10:E75.2] Preregistration [547820]
Company
Amicus Therape.
Structure
Download
2D MOL

3D MOL
Formula
C6H13NO4
Canonical SMILES
C1C(C(C(C(N1)CO)O)O)O
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
InChIKey
LXBIFEVIBLOUGU-DPYQTVNSSA-N
CAS Number
CAS 167933-07-5
PubChem Compound ID
176077
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
Target and Pathway
Target(s) Alpha-galactosidase A Target Info Modulator [533046], [537257]
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
Ref 547820Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800019652)
Ref 533046AT-1001: a high-affinity alpha3beta4 nAChR ligand with novel nicotine-suppressive pharmacology. Br J Pharmacol. 2015 Apr;172(7):1834-45.
Ref 537257The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines. J Inherit Metab Dis. 2009 Jun;32(3):424-40. Epub 2009 Apr 18.

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