Isofagomine tartrate

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Isofagomine tartrate
File:Isofagomine tartrate structure.svg
Systematic (IUPAC) name
(3R,4R,5R)-3,4-Dihydroxy-5-(hydroxymethyl)piperidine L-(+)-tartrate
Clinical data
Routes of
administration		 Oral
Identifiers
CAS Number 957230-65-8
ATC code none
PubChem CID 23581846
Synonyms AT-2101, HGT-3410
Chemical data
Formula C10H19NO9
Molar mass 297.25916 g/mol[[Script error: No such module "String".]]
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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 EMEA.[2]

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.[3][4]

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

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  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. ↑ Dulsat, C., Mealy, N. (2009). "Isofagomine tartrate". Drugs of the Future. 34 (1): 23. doi:10.1358/dof.2009.034.01.1323946. 
  4. ↑ 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): 13813–13818. doi:10.1073/pnas.0605928103. PMC 1564243 Freely accessible. PMID 16945909. 
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