Tafamidis is a disease-modifying drug developed to treat transthyretin amyloidosis (ATTR), a rare condition caused by misfolding and aggregation of transthyretin protein that leads to nerve and heart damage. It was originally discovered and developed by the Japanese company Fujirebio Pharmaceuticals and later advanced through clinical development by Pfizer. Tafamidis works as a transthyretin stabilizer, binding to the TTR protein and preventing its dissociation into amyloid-forming monomers. It was first approved in 2011 in the European Union for transthyretin familial amyloid polyneuropathy and later received broader approval, including for transthyretin amyloid cardiomyopathy in the United States in 2019. Its development marked an important milestone as one of the first targeted therapies addressing the underlying cause of ATTR rather than only treating symptoms.
