Miglustat hydrochloride

Modify Date: 2025-08-25 22:40:16

Miglustat hydrochloride Structure
Miglustat hydrochloride structure
Common Name Miglustat hydrochloride
CAS Number 210110-90-0 Molecular Weight 255.739
Density N/A Boiling Point 421.2ºC at 760 mmHg
Molecular Formula C10H22ClNO4 Melting Point 169-172ºC
MSDS N/A Flash Point 208.5ºC

 Use of Miglustat hydrochloride


Miglustat hydrochloride is an inhibitor of glucosylceramide synthase, primarily to treat Type I Gaucher disease (GD1).Target: OthersMiglustat is an inhibitor of the ceramide-specific glycosyltransferase, which catalyzes the first step of glycosphingolipid biosynthesis and is currently approved for the oral treatment of type 1 GD [1]. Consumption of a standard high-fat breakfast within 30 minutes before administration of miglustat significantly reduced peak exposure but did not significantly affect the extent of systemic exposure to miglustat. The peak plasma concentration (C(max)) decreased by 36% on average following administration with food. Area under the plasma concentration-time curve (AUC(0-infinity)) showed a modest (14%) decrease with food, but the 90% confidence interval was within the acceptance limit of 80% to 125%. The median (min-max) time to C(max) (t(max)) was prolonged from 2.5 (1.0-4.0) hours in the fasted state to 4.5 (1.5-8.0) hours in the fed state, whereas the apparent terminal half-life was approximately 8 hours and not affected by food [2].

 Names

Name N-Butyldeoxynojirimycin Hydrochloride
Synonym More Synonyms

 Miglustat hydrochloride Biological Activity

Description Miglustat hydrochloride is an inhibitor of glucosylceramide synthase, primarily to treat Type I Gaucher disease (GD1).Target: OthersMiglustat is an inhibitor of the ceramide-specific glycosyltransferase, which catalyzes the first step of glycosphingolipid biosynthesis and is currently approved for the oral treatment of type 1 GD [1]. Consumption of a standard high-fat breakfast within 30 minutes before administration of miglustat significantly reduced peak exposure but did not significantly affect the extent of systemic exposure to miglustat. The peak plasma concentration (C(max)) decreased by 36% on average following administration with food. Area under the plasma concentration-time curve (AUC(0-infinity)) showed a modest (14%) decrease with food, but the 90% confidence interval was within the acceptance limit of 80% to 125%. The median (min-max) time to C(max) (t(max)) was prolonged from 2.5 (1.0-4.0) hours in the fasted state to 4.5 (1.5-8.0) hours in the fed state, whereas the apparent terminal half-life was approximately 8 hours and not affected by food [2].
Related Catalog
References

[1]. Abian, O., et al., Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase. Mol Pharm, 2011. 8(6): p. 2390-7.

[2]. van Giersbergen, P.L. and J. Dingemanse, Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol, 2007. 47(10): p. 1277-82.

 Chemical & Physical Properties

Boiling Point 421.2ºC at 760 mmHg
Melting Point 169-172ºC
Molecular Formula C10H22ClNO4
Molecular Weight 255.739
Flash Point 208.5ºC
Exact Mass 255.123734
PSA 84.16000
Vapour Pressure 7.37E-09mmHg at 25°C
InChIKey QPAFAUYWVZMWPR-ZSOUGHPYSA-N
SMILES CCCCN1CC(O)C(O)C(O)C1CO.Cl
Storage condition 2-8℃

 Safety Information

Hazard Codes Xi

 Synonyms

Hydrogen chloride (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)-3,4,5-piperidinetriol (1:1:1)
(2R,3R,4R,5S)-1-Butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride (1:1)
3,4,5-Piperidinetriol, 1-butyl-2-(hydroxymethyl)-, chloride, hydrogen salt, (2R,3R,4R,5S)- (1:1)
Miglustat hydrochloride,(2R,3R,4R,5S)-1-Butyl-2-(hydroxymethyl)-3,4,5-piperidinetriolhydrochloride
Miglustat (hydrochloride)
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