tafamidis

Tafamidis is a small molecule drug used to treat cardiomyopathy caused by wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. It acts as a selective stabilizer of the transthyretin (TTR) protein by binding to its thyroxine-binding sites, thereby stabilizing the tetrameric form and preventing dissociation into monomers—the rate-limiting step in amyloid formation. This mechanism reduces the formation of amyloid deposits in tissues, particularly the heart, slowing disease progression and reducing mortality and hospitalization related to heart failure. Tafamidis was developed by FoldRX and is marketed under the brand names Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis). It was first approved in Europe in 2011 for polyneuropathy associated with transthyretin amyloidosis and later received FDA approval in 2019 for ATTR-CM[1][3][4][5][7].

Brand names

VyndaqelVyndamax

Other names

tafamidis meglumine