Tafamidis free acid form is a non-nonsteroidal anti-inflammatory benzoxazole derivative. Tafamidis, sold under the brand name Vyndamax among others, was developed by FoldRX, now acquired by Pfizer. It was approved by FDA in 2019.
Tafamidis is used as a drug for the treatment of transthyretin-related hereditary amyloidosis as well as familial amyloid polyneuropathy, or FAP, a rare but fatal neurodegenerative disease.
Tafamidis is also a transthyretin (TTR) stabilizer recently approved to slow the neurologic impairment in TTR familial amyloid polyneuropathy (TTR-FAP). The pivotal studies on Tafamidis reported encouraging results on the short term, in the early onset Val30Met-TTR-FAP patients at an early stage of the neuropathy.
Tafamidis was approved for use in the European Union by the European Medicines Agency in November 2011, specifically for the treatment of early stage transthyretin-related hereditary amyloidosis or familial amyloid polyneuropathy or FAP (all mutations).
Tafamidis is also approved for use in Brazil, Argentina, Mexico and Israel by the relevant authorities.
It is also indicated for treatment of cardiomyopathy in adults. In most cases, cardiomyopathy causes the heart muscle to become enlarged, thick or rigid. In rare instances, diseased heart muscle tissue is replaced with scar tissue.