Isofagomine

Afegostat

Clinical data
Trade names	Plicera
Routes of administration	Oral
ATC code	none
Legal status
Legal status	Development terminated
Identifiers
IUPAC name (3R,4R,5R)-5-(Hydroxymethyl)-3,4-piperidinediol
Synonyms	Isofagomine; AT-2101, HGT-3410
CAS Number	169105-89-9 Y
PubChem CID	447607
IUPHAR/BPS	7410
ChemSpider	394649
UNII	G23AP190YS
KEGG	D09576
ChEMBL	CHEMBL206468
Chemical and physical data
Formula	C6H13NO3
Molar mass	147.17 g/mol
3D model (JSmol)	Interactive image
SMILES C1[C@@H]([C@H]([C@@H](CN1)O)O)CO
InChI InChI=1S/C6H13NO3/c8-3-4-1-7-2-5(9)6(4)10/h4-10H,1-3H2/t4-,5-,6-/m1/s1 Key:QPYJXFZUIJOGNX-HSUXUTPPSA-N

Afegostat (INN; also known as isofagomine; planned trade name Plicera) was an experimental drug for the treatment of certain forms of Gaucher's disease, that was being developed by Amicus Therapeutics and Shire plc until a failed clinical trial in 2009 led to termination of its development. The substance was used in form of the tartrate.

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. Afegostat, an iminosugar, binds selectively to N370S glucocerebrosidase and restores its correct conformation and, consequently, enhances its activity about threefold.

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.