Isofagomine

(BAN)
Isofagomine Chemical formula

Chemical information

AT-2101 (tartrate). (3R,4R,5R)-3,4-Dihydroxy-5-(hydroxymethyl)piperidine.
Chemical formula: C6H13NO3 = 147.2.
CAS — 169105-89-9 (isofagomine); 161302-93-8 (isofagomine hydrochloride).
NOTE. The code AT-2101 has also been applied to a topical formulation of diclofenac in hyaluronic acid used in the treatment of actinic keratoses.

Profile

Isofagomine is an iminosugar under investigation as the tartrate for oral therapy of Gaucher disease. It is a pharmacological chaperone that stabilises the variant lysosomal glucocerebrosidase facilitating its folding and transport from the endoplasmic reticulum into lysosomes, thereby increasing the pool of active endogenous enzyme.
Published May 08, 2019.