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

MedKoo CAT#: 510317

CAS#: 957230-65-8 (tartrate)

Description: Afegostat, aslo known as Isofagomine, and AT2101, was an experimental drug for the treatment of certain forms of Gaucher's disease. Isofagomine (IFG) is an acid β-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. FG inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes (N370S and V394L). Administration of IFG (30 mg/kg/day) to the mice homozygous for GCase mutations (V394L, D409H, or D409V) led to increased GCase activity in visceral tissues and brain extracts.

Chemical Structure

CAS# 957230-65-8 (tartrate)

Theoretical Analysis

MedKoo Cat#: 510317
Name: Afegostat
CAS#: 957230-65-8 (tartrate)
Chemical Formula: C10H19NO9
Exact Mass:
Molecular Weight: 297.26
Elemental Analysis: C, 40.41; H, 6.44; N, 4.71; O, 48.44

Price and Availability

Size Price Availability Quantity
5.0mg USD 190.0 Same Day
10.0mg USD 350.0 Same Day
25.0mg USD 550.0 Same Day
50.0mg USD 950.0 Same Day
100.0mg USD 1650.0 Same Day
200.0mg USD 2650.0 Same Day
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Related CAS #: 957230-65-8 (tartrate)   169105-89-9 (free base)   161302-93-8 (HCl)   1084896-48-9 (acetate)   1084896-50-3 (TFA)    

Synonym: AT2101; AT-2101, AT 2101; Isofagomine D-Tartrate; Isofagomine; Afegostat; D-Isofagomine; isoFagomine. brand name: Plicera

IUPAC/Chemical Name: (3R,4R,5R)-5-(hydroxymethyl)piperidine-3,4-diol (2S,3S)-2,3-dihydroxysuccinate.


InChi Code: InChI=1S/C6H13NO3.C4H6O6/c8-3-4-1-7-2-5(9)6(4)10;5-1(3(7)8)2(6)4(9)10/h4-10H,1-3H2;1-2,5-6H,(H,7,8)(H,9,10)/t4-,5-,6-;1-,2-/m10/s1

SMILES Code: O[C@@H]1CNC[C@H](CO)[C@H]1O.O=C(O)[C@@H](O)[C@H](O)C(O)=O

Appearance: Brown solid powder (highly hydroscopic, may become oil in air).

Purity: >98% (or refer to the Certificate of Analysis), supplied as ethanol-water solution (1:1, v/v) with isofagomine D-Tartrate concentrion = 100 mg/mL.

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 water and ethanol.

Shelf Life: >5 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

Preparing Stock Solutions

The following data is based on the product molecular weight 297.26 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

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1: Front S, Almeida S, Zoete V, Charollais-Thoenig J, Gallienne E, Marmy C, Pilloud V, Marti R, Wood T, Martin OR, Demotz S. 4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations. Bioorg Med Chem. 2018 Nov 1;26(20):5462-5469. doi: 10.1016/j.bmc.2018.09.023. Epub 2018 Sep 21. PubMed PMID: 30270003.

2: Lebl R, Thonhofer M, Tysoe C, Pabst BM, Schalli M, Weber P, Paschke E, Stütz AE, Tschernutter M, Windischhofer W, Withers SG. A Morita-Baylis-Hillman based route to C-5a-chain-extended 4-epi-isofagomine type glycosidase inhibitors. Carbohydr Res. 2017 Apr 10;442:31-40. doi: 10.1016/j.carres.2017.03.003. Epub 2017 Mar 6. PubMed PMID: 28288345.

3: Front S, Biela-Banaś A, Burda P, Ballhausen D, Higaki K, Caciotti A, Morrone A, Charollais-Thoenig J, Gallienne E, Demotz S, Martin OR. (5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B. Eur J Med Chem. 2017 Jan 27;126:160-170. doi: 10.1016/j.ejmech.2016.09.095. Epub 2016 Sep 29. PubMed PMID: 27750150.

4: Thonhofer M, Weber P, Gonzalez Santana A, Tysoe C, Fischer R, Pabst BM, Paschke E, Schalli M, Stütz AE, Tschernutter M, Windischhofer W, Withers SG. Synthesis of C-5a-substituted derivatives of 4-epi-isofagomine: notable β-galactosidase inhibitors and activity promotors of GM1-gangliosidosis related human lysosomal β-galactosidase mutant R201C. Carbohydr Res. 2016 Jun 24;429:71-80. doi: 10.1016/j.carres.2016.03.020. Epub 2016 Mar 31. PubMed PMID: 27063389.

5: Thonhofer M, Weber P, Santana AG, Fischer R, Pabst BM, Paschke E, Schalli M, Stütz AE, Tschernutter M, Windischhofer W, Withers SG. Synthesis of C-5a-chain extended derivatives of 4-epi-isofagomine: Powerful β-galactosidase inhibitors and low concentration activators of GM1-gangliosidosis-related human lysosomal β-galactosidase. Bioorg Med Chem Lett. 2016 Mar 1;26(5):1438-42. doi: 10.1016/j.bmcl.2016.01.059. Epub 2016 Jan 22. PubMed PMID: 26838810.

6: Thonhofer M, Gonzalez Santana A, Fischer R, Torvisco Gomez A, Saf R, Schalli M, Stütz AE, Withers SG. 5-Fluoro derivatives of 4-epi-isofagomine as D-galactosidase inhibitors and potential pharmacological chaperones for GM1-gangliosidosis as well as Fabry's disease. Carbohydr Res. 2016 Feb;420:6-12. doi: 10.1016/j.carres.2015.10.009. Epub 2015 Dec 8. PubMed PMID: 26717544.

7: Lindbäck E, Laursen BW, Poulsen JC, Kilså K, Pedersen CM, Bols M. A fluorescence study of isofagomine protonation in β-glucosidase. Org Biomol Chem. 2015 Jun 21;13(23):6562-6. doi: 10.1039/c5ob00624d. Epub 2015 May 15. PubMed PMID: 25978843.

8: Lahiri R, Palanivel A, Kulkarni SA, Vankar YD. Synthesis of isofagomine-pyrrolidine hybrid sugars and analogues of (-)-steviamine and (+)-hyacinthacine C5 using 1,3-dipolar cycloaddition reactions. J Org Chem. 2014 Nov 21;79(22):10786-800. doi: 10.1021/jo5016745. Epub 2014 Nov 4. PubMed PMID: 25333970.

9: Reddy YS, Kancharla PK, Roy R, Vankar YD. Aza-Claisen rearrangement of 2-C-hydroxymethyl glycals as a versatile strategy towards synthesis of isofagomine and related biologically important azasugars. Org Biomol Chem. 2012 Apr 14;10(14):2760-73. doi: 10.1039/c2ob06851f. Epub 2012 Feb 27. PubMed PMID: 22371151.

10: Malik G, Guinchard X, Crich D. Asymmetric synthesis of polyhydroxylated N-alkoxypiperidines by ring-closing double reductive amination: facile preparation of isofagomine and analogues. Org Lett. 2012 Jan 20;14(2):596-9. doi: 10.1021/ol203213f. Epub 2011 Dec 23. PubMed PMID: 22195980.

11: Sun Y, Liou B, Xu YH, Quinn B, Zhang W, Hamler R, Setchell KD, Grabowski GA. Ex vivo and in vivo effects of isofagomine on acid β-glucosidase variants and substrate levels in Gaucher disease. J Biol Chem. 2012 Feb 3;287(6):4275-87. doi: 10.1074/jbc.M111.280016. Epub 2011 Dec 13. PubMed PMID: 22167193; PubMed Central PMCID: PMC3281716.

12: Moréra S, Vigouroux A, Stubbs KA. A potential fortuitous binding of inhibitors of an inverting family GH9 β-glycosidase derived from isofagomine. Org Biomol Chem. 2011 Sep 7;9(17):5945-7. doi: 10.1039/c1ob05766a. Epub 2011 Jul 25. PubMed PMID: 21785782.

13: Lindbäck E, López O, Fernández-Bolaños JG, Sauer SP, Bols M. An isofagomine analogue with an amidine at the pseudoanomeric position. Org Lett. 2011 Jun 3;13(11):2908-11. doi: 10.1021/ol200942g. Epub 2011 May 6. PubMed PMID: 21548609.

14: Kato A, Miyauchi S, Kato N, Nash RJ, Yoshimura Y, Nakagome I, Hirono S, Takahata H, Adachi I. Docking and SAR studies of D- and L-isofagomine isomers as human β-glucocerebrosidase inhibitors. Bioorg Med Chem. 2011 Jun 1;19(11):3558-68. doi: 10.1016/j.bmc.2011.04.011. Epub 2011 Apr 13. PubMed PMID: 21546253.

15: Sun Y, Ran H, Liou B, Quinn B, Zamzow M, Zhang W, Bielawski J, Kitatani K, Setchell KD, Hannun YA, Grabowski GA. Isofagomine in vivo effects in a neuronopathic Gaucher disease mouse. PLoS One. 2011 Apr 20;6(4):e19037. doi: 10.1371/journal.pone.0019037. PubMed PMID: 21533102; PubMed Central PMCID: PMC3080394.

16: Khanna R, Benjamin ER, Pellegrino L, Schilling A, Rigat BA, Soska R, Nafar H, Ranes BE, Feng J, Lun Y, Powe AC, Palling DJ, Wustman BA, Schiffmann R, Mahuran DJ, Lockhart DJ, Valenzano KJ. The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase. FEBS J. 2010 Apr;277(7):1618-38. doi: 10.1111/j.1742-4658.2010.07588.x. Epub 2010 Feb 10. PubMed PMID: 20148966; PubMed Central PMCID: PMC2874831.

17: Kornhaber GJ, Tropak MB, Maegawa GH, Tuske SJ, Coales SJ, Mahuran DJ, Hamuro Y. Isofagomine induced stabilization of glucocerebrosidase. Chembiochem. 2008 Nov 3;9(16):2643-9. doi: 10.1002/cbic.200800249. PubMed PMID: 18932186; PubMed Central PMCID: PMC2910746.

18: Shen JS, Edwards NJ, Hong YB, Murray GJ. Isofagomine increases lysosomal delivery of exogenous glucocerebrosidase. Biochem Biophys Res Commun. 2008 May 16;369(4):1071-5. doi: 10.1016/j.bbrc.2008.02.125. Epub 2008 Mar 6. PubMed PMID: 18328804; PubMed Central PMCID: PMC2374924.

19: Steet R, Chung S, Lee WS, Pine CW, Do H, Kornfeld S. Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase. Biochem Pharmacol. 2007 May 1;73(9):1376-83. Epub 2006 Dec 15. PubMed PMID: 17217920; PubMed Central PMCID: PMC1892903.

20: Yu Z, Sawkar AR, Whalen LJ, Wong CH, Kelly JW. Isofagomine- and 2,5-anhydro-2,5-imino-D-glucitol-based glucocerebrosidase pharmacological chaperones for Gaucher disease intervention. J Med Chem. 2007 Jan 11;50(1):94-100. PubMed PMID: 17201413; PubMed Central PMCID: PMC2543937.

Additional Information

Afegostat was invented by Mikael Bols and Troels Skrydstrup, and was first prepared by Jespersen and Bols. Amicus Therapeutics licensed patents related to afegostat from Mt. Sinai School of Medicine, University of Maryland, and Novo Nordisk A/S, and also signed a collaboration agreement with Shire plc related to this drug and others. It was granted orphan drug status by the European Medicines Agency (EMA) and by the US FDA. When afegostat failed a Phase II clinical trial in 2009, Shire terminated the collaboration agreement and Amicus determined it would no longer develop the afegostat. The first patents in Amicus' patent portfolio on afegostat expire in 2015. (source: