afegostat afegostat 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. afegostatclinical datatrade namespliceraother namesisofagomine; at-2101, hgt-3410routes ofadministrationoralatc codenonelegal statuslegal status development terminated identifiers iupac name (3r,4r,5r)-5-(hydroxymethyl)-3,4-piperidinediol cas number169105-89-9 ypubchem cid447607iuphar/bps7410chemspider394649uniig23ap190yskeggd09576chemblchembl206468comptox dashboard (epa)dtxsid20168651 chemical and physical dataformulac6h13no3molar mass147.174 g·mol−13d model (jsmol)interactive image smiles c1(((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/s1key:qpyjxfzuijognx-hsuxutppsa-n (verify) 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. afegostat, an iminosugar, binds selectively to n370s glucocerebrosidase and restores its correct conformation and, consequently, enhances its activity about threefold. invention and development 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. they also signed a collaboration agreement with shire plc related to this drug and others.: 13 it was granted orphan drug status by the european medicines agency (ema) and by the us fda.: 10 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.: 2, 10, 11 the first patents in amicus' patent portfolio on afegostat expired in 2015.: 12