Migalastat hydrochloride

Modify Date: 2024-01-03 05:39:54

Migalastat hydrochloride Structure
Migalastat hydrochloride structure
Common Name Migalastat hydrochloride
CAS Number 75172-81-5 Molecular Weight 199.63300
Density N/A Boiling Point 382.7ºC at 760 mmHg
Molecular Formula C6H14ClNO4 Melting Point 260ºC
MSDS USA Flash Point 185.2ºC

 Use of Migalastat hydrochloride


Migalastat hydrochloride (1-Deoxygalactonojirimycin hydrochloride) is a potent and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM for human α-Gal A.

 Names

Name deoxygalactonojirimycin, hydrochloride
Synonym More Synonyms

 Migalastat hydrochloride Biological Activity

Description Migalastat hydrochloride (1-Deoxygalactonojirimycin hydrochloride) is a potent and competitive inhibitor of α-galactosidase A (α-Gal A) with an IC50 of 0.04 μM for human α-Gal A.
Related Catalog
Target

IC50: 0.04 μM (human α-Gal A)[1];Ki: 0.04 μM (human α-Gal A)[1]

In Vitro Both IC50 and Ki values of migalastat hydrochloride toward human lysosomal a-Gal A are 0.04 μM[1].
In Vivo Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A. α-Gal A activity in heart, kidney, spleen, and liver is increased dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A with migalastat hydrochloride treatment. The half-life of DGJ is less than 1 day in all major issues and that of the enzyme synthesized during the DGJ treatment period is approximately 4 days[2]. Oral administration of migalastat hydrochloride reduces tissue GL-3 in fabry transgenic mice, and in urine and kidneys of some FD patients. Oral administration of migalastat hydrochloride to transgenic mice reduces elevated lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively, generally equal to or greater than observed for GL-3[3].
Animal Admin Mice: Migalastat hydrochloride is dissolved in drinking water. Age-matched male homozygous TgM, TgM/KO, and nontransgenic (Non-Tg) C57BL/6 mice are supplied drinking water containing DGJ ad libitum. Body weight is monitored weekly during the DGJ treatment period. Average daily DGJ dosage is estimated based on the consumption of drinking water. Mice fed drinking water containing DGJ at 0.05 mM typically received DGJ at approximately 3 mg/kg body weight/day[2].
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.

[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.

[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.

 Chemical & Physical Properties

Boiling Point 382.7ºC at 760 mmHg
Melting Point 260ºC
Molecular Formula C6H14ClNO4
Molecular Weight 199.63300
Flash Point 185.2ºC
Exact Mass 199.06100
PSA 92.95000

 Safety Information

Personal Protective Equipment dust mask type N95 (US);Eyeshields;Gloves
Hazard Codes Xi
Risk Phrases R36/37/38
Safety Phrases 26-36
RIDADR NONH for all modes of transport

 Articles1

More Articles
Paulsen, H., et al.

Chem. Ber. 113 , 2601, (1980)

 Synonyms

(1R,3R,4R,5S)-3,4,5-trihydroxy-2-(hydroxymethyl)piperidinium chloride
1,5-dideoxy-1,5-imino-D-glucitol hydrochloride
1-Deoxymannojirimycinehydrochloride
DEOXYMANNONOJIRIMYCIN HCL
(+)-(2R,3R,4R,5S)-2-hydroxymethyl-3,4,5-trihydroxypiperidine hydrochloride
DEOXYMANNOJIRIMYCIN,HYDROCHLORIDE
(+)-1-deoxynojirimycin hydrochloride
1-DEOXYMANNOJIRIMYCIN HCL
DMM
DEOXYMANNOJIRIMYCIN HCL
DMJ,HYDROCHLORIDE
1-DeoxymannojirimycineHCl
Migalastat (hydrochloride)
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