Systematic (IUPAC) name | |
---|---|
(3R,4R,5R)-3,4-Dihydroxy-5-(hydroxymethyl)piperidine L-(+)-tartrate | |
Clinical data | |
Pregnancy cat. | ? |
Legal status | ? |
Routes | Oral |
Identifiers | |
CAS number | 957230-65-8 |
ATC code | None |
PubChem | CID 23581846 |
Synonyms | AT-2101, HGT-3410 |
Chemical data | |
Formula | C10H19NO9 |
Mol. mass | 297.25916 g/mol |
SMILES | eMolecules & PubChem |
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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 European Medicines Agency (EMA).[2] Isofagomine way invented Mikael Bols and Troels Skrydstrup, and was first prepared by Jespersen and Bols.[3]
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]