VX-814

VX-814 is a small molecule corrector developed by Vertex Pharmaceuticals for the treatment of alpha-1 antitrypsin deficiency (AATD), a genetic disorder characterized by misfolding and accumulation of the alpha-1 antitrypsin protein in the liver. The drug was designed to act as an alpha 1-antitrypsin modulator, correcting protein misfolding and facilitating proper folding and secretion of functional AAT from hepatocytes. This mechanism aimed to increase circulating levels of functional AAT, potentially reducing liver damage and improving lung function in affected patients. Development was discontinued after Phase II clinical trials due to safety concerns, specifically elevated liver enzymes that prevented safe achievement of target exposure levels[1][2][3][6][8].