| Identification | Back Directory | [Name]
DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE | [CAS]
108147-54-2 | [Synonyms]
DGJ
Migalasta
MIGALASTAT
GALACTOSTATIN HCL
DGJ, HYDROCHLORIDE
1-Deoxygalactonojirimycin
DEOXYGALACTONOJIRIMYCIN HCL
1-DEOXYGALACTONOJIRIMYCIN HCL
1,5-dideoxy-1,5-iminogalactitol
DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE
1,5-DIDEOXY-1,5-IMINO-D-GALACTITOL, HYDROCHLORIDE
(2R,3S,4R,5S)-2-(Hydroxymethyl)-3,4,5-piperidinetriol
(2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, (2R,3S,4R,5S)- | [Molecular Formula]
C6H13NO4 | [MDL Number]
MFCD00269962 | [MOL File]
108147-54-2.mol | [Molecular Weight]
163.17 |
| Chemical Properties | Back Directory | [Melting point ]
243-245 °C | [Boiling point ]
361.1±42.0 °C(Predicted) | [density ]
1.456±0.06 g/cm3(Predicted) | [storage temp. ]
2-8°C | [pka]
13.77±0.70(Predicted) |
| Hazard Information | Back Directory | [Uses]
Migalastat (GR181413A free base) is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity[1]. | [Definition]
ChEBI: Migalastat is a member of piperidines. | [Brand name]
Treatment of Fabry
disease. | [in vivo]
Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A[2].
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM)[2].
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment[2].
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively[3]. | Animal Model: | Male nontransgenic (Non-Tg) C57BL/6 mice; transgenic mice expressing human mutant R301Q α-Gal A (TgM), α-Gal A knockout mice (KO), mice express human R301Q α-Gal A in a null background (TgM/KO)[2] | | Dosage: | 3 mg/kg | | Administration: | Oral gavage; every day for 4 weeks | | Result: | Reduced Globotriaosylceramide (Gb3) storage remarkably in kidney of mice. |
| [References]
[1] Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267(13):4179-86. DOI:10.1046/j.1432-1327.2000.01457.x
[2] Ishii S, et al. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328(3):723-31. DOI:10.1124/jpet.108.149054
[3] Young-Gqamana B, et al. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. PLoS One. 2013;8(3):e57631. DOI:10.1371/journal.pone.0057631
[4] Welford RWD, et al. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct. 27(19):3392-3403. DOI:10.1093/hmg/ddy248 |
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