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MedKoo Cat#: 564791 | Name: Miglustat

Description:

WARNING: This product is for research use only, not for human or veterinary use.

Miglustat, also known as OGT 918, is a drug developed by Actelion and is used primarily to treat Type I Gaucher disease (GD1). It is marketed under the trade name Zavesca. Miglustat is an imino sugar, a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste. 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).

Chemical Structure

Miglustat
Miglustat
CAS#72599-27-0 (free base)

Theoretical Analysis

MedKoo Cat#: 564791

Name: Miglustat

CAS#: 72599-27-0 (free base)

Chemical Formula: C10H21NO4

Exact Mass: 219.1471

Molecular Weight: 219.28

Elemental Analysis: C, 54.77; H, 9.65; N, 6.39; O, 29.18

Price and Availability

This product is currently not in stock but may be available through custom synthesis. To ensure cost efficiency, the minimum order quantity is 1 gram. The estimated lead time is 2 to 4 months, with pricing dependent on the complexity of the synthesis (typically high for intricate chemistries). Quotes for quantities below 1 gram will not be provided. To request a quote, please click the button below. Note: If this product becomes available in stock in the future, pricing will be listed accordingly.
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Related CAS #
72599-27-0 (free base) 210110-90-0 (HCl)
Synonym
OGT918; OGT 918; OGT-918; Miglustat; N-butyldeoxynojirimycin; NB-DNJ; N-Butylmoranoline; Miglustat hydrochloride; Brand name: Zavesca.
IUPAC/Chemical Name
(2R,3R,4R,5S)-1-Butyl-2-(hydroxymethyl)piperidine-3,4,5-triol
InChi Key
UQRORFVVSGFNRO-UTINFBMNSA-N
InChi Code
InChI=1S/C10H21NO4/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H3/t7-,8+,9-,10-/m1/s1
SMILES Code
O[C@@H]1[C@@H](CO)N(CCCC)C[C@H](O)[C@H]1O
Appearance
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
Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility
Soluble in DMSO
Shelf Life
>3 years if stored properly
Drug Formulation
This drug may be formulated in DMSO
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
More Info

Preparing Stock Solutions

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.

Recalculate based on batch purity %
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(®)) 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, Chérin 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.