Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of α-Gal A. Oral administration of migalastat HCl reduces tissue GL-3 in Fabry transgenic mice, and in urine and kidneys of some FD patients. Migalastat HCl may provide a potential novel genotype-specific treatment for Fabry Disease (FD). Phase 3 studies are ongoing. Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues.
| Catalog Number | R020682 |
| CAS Number | 75172-81-5 |
| Synonyms | (2R,3S,4R,5S)-2-Hydroxymethyl-3,4,5-piperidinetriol Hydrochloride; Migalastat Hydrochloride; 1-Deoxygalactostatin Hydrochloride; |
| Molecular Formula | C6H13NO4 • HCl |
| Purity | 95% |
| Target | Galactosidase |
| Solubility | Soluble in DMSO |
| Storage | Store at -20°C |
| InChIKey | ZJIHMALTJRDNQI-OLALXQGDSA-N |
| Reference | </br>1:A Phase 2 study of migalastat hydrochloride in females with Fabry disease: selection of population, safety and pharmacodynamic effects. Giugliani R, Waldek S, Germain DP, Nicholls K, Bichet DG, Simosky JK, Bragat AC, Castelli JP, Benjamin ER, Boudes PF.Mol Genet Metab. 2013 May;109(1):86-92. doi: 10.1016/j.ymgme.2013.01.009. Epub 2013 Jan 26. PMID: 23474038 Free Article</br>2:Pharmacokinetics, safety, and tolerability following single-dose migalastat hydrochloride (GR181413A/AT1001) in healthy male Japanese subjects. Ino H, Takahashi N, Terao T, Mudd PN Jr, Hirama T.J Drug Assess. 2013 Jul 24;2(1):87-93. doi: 10.3109/21556660.2013.827117. eCollection 2013. PMID: 27536442 Free PMC Article |
