Title: Miglustat
CAS Registry Number: 72599-27-0
CAS Name: (2R,3R,4R,5S)-1-Butyl-2-(hydroxymethyl)-3,4,5-piperidinetriol
Additional Names: N-butyldeoxynojirimycin; N-butylmoranoline
Manufacturers' Codes: OGT-918; SC-48334
Trademarks: Zavesca (Actelion)
Molecular Formula: C10H21NO4
Molecular Weight: 219.28
Percent Composition: C 54.77%, H 9.65%, N 6.39%, O 29.19%
Literature References: Amino sugar; inhibitor of glucosyltransferase, an enzyme involved in the biosynthesis of glycosphingolipids. Prepn: B. Junge et al., DE 2758025; see also, eidem, US 4639436 (1979, 1987 both to Bayer). Improved synthesis: E. W. Baxter, A. B. Reitz, J. Org. Chem. 59, 3175 (1994); C. R. R. Matos et al., Synthesis 1999, 571. In vitro efficacy vs HIV: A. Karpas et al., Proc. Natl. Acad. Sci. USA 85, 9229 (1988). Inhibition of glycolipid biosynthesis: F. M. Platt et al., J. Biol. Chem. 269, 8362 (1994). Clinical evaluation in Gaucher's disease: T. Cox et al., Lancet 355, 1481 (2000). Review of pharmacology and clinical development in Gaucher's disease: P. L. McCormack, K. L. Goa, Drugs 63, 2427-2434 (2003).
Properties: White to off-white crystalline solid, mp 125-126°. [a]D25 -15.9° (c = 0.77 in water). Highly sol in water (>1000 mg/ml).
Melting point: mp 125-126°
Optical Rotation: [a]D25 -15.9° (c = 0.77 in water)
Therap-Cat: In treatment of inherited glycosphingolipid lysosomal storage disorders. |