Isofagomine tartrate

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Isofagomine tartrate
Systematic (IUPAC) name
(3R,4R,5R)-3,4-Dihydroxy-5-(hydroxymethyl)piperidine L-(+)-tartrate
Clinical data
Legal status ?
Routes Oral
Identifiers
CAS number 957230-65-8 YesY
ATC code None
PubChem CID 23581846
Synonyms AT-2101, HGT-3410
Chemical data
Formula C10H19NO9 
Mol. mass 297.25916 g/mol
 YesY (what is this?)  (verify)

Isofagomine tartrate (planned trade name Plicera) is an experimental drug for the treatment of certain forms of Gaucher's disease, developed by Amicus Therapeutics and Shire plc.[1] It has been granted orphan drug status by the European Medicines Agency (EMA).[2] Isofagomine was invented by Mikael Bols and Troels Skrydstrup, and was first prepared by Jespersen and Bols.[3]

Mechanism of action

β-glucocerebrosidase, an enzyme needed for the metabolisation of glucocerebroside, is misfolded in individuals with Gaucher's disease due to various mutations, one of which is called N370S. Isofagomine binds selectively to N370S glucocerebrosidase and restores its correct conformation and, consequently, enhances its activity about threefold.[4][5]

See also

  • Imiglucerase, a recombinant human β-glucocerebrosidase for enzyme replacement therapy of Gaucher's disease
  • Miglustat, another orphan drug for the treatment of Gaucher's disease with a different mechanism of action

References

  1. ↑ H. Spreitzer (20 June 2009). "Neue Wirkstoffe - Isofagomin tartrat". Österreichische Apothekerzeitung (in German) (13/2009): 736. 
  2. ↑ Public summary of positive opinion for orphan designation of isofagomine tartrate for the treatment of Gaucher disease
  3. ↑ Jespersen, T. M.; Dong, W; Skrydstrup, T; Sierks, M.R; Lundt, I; Bols, M. (1994). "Isofagomine, a Potent New Glycosidase Inhibitor". Angew. Chem. Int. Ed. Engl. 33 (17): 1778–1779. doi:10.1002/anie.199417781. 
  4. ↑ Dulsat, C., Mealy, N. (2009). "Isofagomine tartrate". Drugs of the Future 34 (1): 23. doi:10.1358/dof.2009.034.01.1323946. 
  5. ↑ Richard A. Steet, Stephen Chung, Brandon Wustman, Allan Powe, Hung Do, and Stuart A. Kornfeld (2006). "The iminosugar isofagomine increases the activity of N370S mutant acid β-glucosidase in Gaucher fibroblasts by several mechanisms". PNAS 103 (37): 13813–8. doi:10.1073/pnas.0605928103. PMC 1564243. PMID 16945909. 
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