WARNING: This product is for research use only, not for human or veterinary use.
MedKoo CAT#: 510249
CAS#: 210110-90-0 (HCl)
Description: Miglustat, also known as OGT 918 and NB-DNJ, is a drug developed by Actelion and is used primarily to treat Type I Gaucher disease (GD1). Miglustat inhibits glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids (it forms glucosylceramide and accumulates within the macrophages). Miglustat is used to treat adults with mild-to-moderate Type I Gaucher disease and it is the first treatment to be approved for patients with NiemannÂ–Pick type C disease
MedKoo Cat#: 510249
Name: Miglustat HCl
CAS#: 210110-90-0 (HCl)
Chemical Formula: C10H21NO4
Molecular Weight: 219.28
Elemental Analysis: C, 46.96; H, 8.67; Cl, 13.86; N, 5.48; O, 25.02
Synonym: OGT918; OGT 918; OGT-918; Miglustat; N-butyldeoxynojirimycin; NB-DNJ; Miglustat hydrochloride; Brand name: Zavesca.
IUPAC/Chemical Name: (2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride
InChi Key: QPAFAUYWVZMWPR-ZSOUGHPYSA-N
InChi Code: InChI=1S/C10H21NO4.ClH/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12;/h7-10,12-15H,2-6H2,1H3;1H/t7-,8+,9-,10-;/m1./s1
SMILES Code: O[C@@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O.[H]Cl
Appearance: white solid powder
Purity: >98% (or refer to the Certificate of Analysis)
Shipping Condition: Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition: 0 Â– 4 C for short term (weeks to 1 month) or -20 C for long terms (months to years).
Solubility: Soluble in water
Shelf Life: >2 years if stored properly
Drug Formulation: This drug may be formulated in water
Stock Solution Storage: 0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code: 2934.99.9001
The following data is based on the product molecular weight 219.28 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
|Concentration / Solvent Volume / Mass||1 mg||5 mg||10 mg|
|1 mM||1.15 mL||5.76 mL||11.51 mL|
|5 mM||0.23 mL||1.15 mL||2.3 mL|
|10 mM||0.12 mL||0.58 mL||1.15 mL|
|50 mM||0.02 mL||0.12 mL||0.23 mL|
1: Kitatani T, Takahashi S, Ikenoya S. [Pharmacological and clinical profiles of miglustat (Brazaves(Â®)) for the treatment of Niemann-Pick type C disease]. Nihon Yakurigaku Zasshi. 2013 Mar;141(3):160-7. Review. Japanese. PubMed PMID: 23470482.
2: Walterfang M, Chien YH, Imrie J, Rushton D, Schubiger D, Patterson MC. Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat. Orphanet J Rare Dis. 2012 Oct 6;7:76. doi: 10.1186/1750-1172-7-76. Review. PubMed PMID: 23039766; PubMed Central PMCID: PMC3552828.
3: Venier RE, Igdoura SA. Miglustat as a therapeutic agent: prospects and caveats. J Med Genet. 2012 Sep;49(9):591-7. doi: 10.1136/jmedgenet-2012-101070. Epub 2012 Aug 14. Review. PubMed PMID: 22892202.
4: Belmatoug N, Burlina A, Giraldo P, Hendriksz CJ, Kuter DJ, Mengel E, Pastores GM. Gastrointestinal disturbances and their management in miglustat-treated patients. J Inherit Metab Dis. 2011 Oct;34(5):991-1001. doi: 10.1007/s10545-011-9368-7. Epub 2011 Jul 21. Review. PubMed PMID: 21779792.
5: Pastores GM, Giraldo P, ChÃ©rin P, Mehta A. Goal-oriented therapy with miglustat in Gaucher disease. Curr Med Res Opin. 2009 Jan;25(1):23-37. doi: 10.1185/03007990802576518 . Review. PubMed PMID: 19210136.
6: Pastores GM. Miglustat: substrate reduction therapy for lysosomal storage disorders associated with primary central nervous system involvement. Recent Pat CNS Drug Discov. 2006 Jan;1(1):77-82. Review. PubMed PMID: 18221193.
7: Lachmann RH. Miglustat: substrate reduction therapy for glycosphingolipid lysosomal storage disorders. Drugs Today (Barc). 2006 Jan;42(1):29-38. Review. PubMed PMID: 16511609.
8: Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. Review. PubMed PMID: 16247743.
9: McCormack PL, Goa KL. Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. Review. PubMed PMID: 14609352.
10: Cox TM, Aerts JM, Andria G, Beck M, Belmatoug N, Bembi B, Chertkoff R, Vom Dahl S, Elstein D, Erikson A, Giralt M, Heitner R, Hollak C, Hrebicek M, Lewis S, Mehta A, Pastores GM, Rolfs A, Miranda MC, Zimran A; Advisory Council to the European Working Group on Gaucher Disease. The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement. J Inherit Metab Dis. 2003;26(6):513-26. Review. PubMed PMID: 14605497.
11: Lachmann RH. Miglustat. Oxford GlycoSciences/Actelion. Curr Opin Investig Drugs. 2003 Apr;4(4):472-9. Review. PubMed PMID: 12808890.
12: Pastores GM, Barnett NL. Substrate reduction therapy: miglustat as a remedy for symptomatic patients with Gaucher disease type 1. Expert Opin Investig Drugs. 2003 Feb;12(2):273-81. Review. PubMed PMID: 12556220.
Miglustat may only be used in the treatment of Type I Gaucher patients for whom enzyme replacement therapy is unsuitable and it's been approved in the European Union for the treatment of progressive neurological manifestations in adult or pediatric patients with NiemannÂ–Pick type C disease (NPC). It has also been approved for NPC treatment in Australia, Brazil, Canada, Israel, Russia, Switzerland and Turkey but not in the United States. (copied from http://en.wikipedia.org/wiki/Miglustat).
The primary pharmacological activity of miglustat is inhibition of the enzyme glucosylceramide synthase, catalyzing the first step in the biosynthesis of glycosphingolipids (GSL), i.e., the formation of glucosylceramide (GlcCer). Reduced formation of GlcCer will lead to decreased biosynthesis of more complex GSL. This therapeutic principle, called substrate reduction therapy (SRT), may be useful in disorders of intracellular (predominantly lysosomal) accumulation of GSL either due to their deficient breakdown or intracellular transport/trafficking. Miglustat exhibits a large volume of distribution and has the capacity to access deep organs such as the brain, bone and lung. (copied from http://en.wikipedia.org/wiki/Miglustat).