Tafamidis (594839-88-0) is a potent and selective transthyretin (TTR) kinetic stabilizer. It binds selectively and with negative cooperativity to the two normally unoccupied thyroxine binding sites of the TTR tetramer (Kd = ~2 and ~200 nM)1. Stabilizes wild-type and mutant TTR under varying conditions in vitro (EC50=2.7-3.2 μM).1 Clinically useful for the treatment of familial amyloid polyneuropathy2,3 and TTR-related cardiomyopathy4.
1) Bulawa et al. (2012), Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits amyloid cascade; Proc. Natl. Acad. Sci. USA, 109 9629
2) Scott (2014), Tafamidis: a review of its use in familial amyloid polyneuropathy; Drugs, 74 1371
3) Zhao et al. (2019), Tafamidis, a Noninvasive Therapy for Delaying Transthyretin Familial Amyloid Polyneuropathy: Systemic Review and Meta-analysis; J. Clin. Neurol., 15 108
4) Lorenzini and Elliott (2019), Tafamidis for the treatment of transthyretin amyloidosis; Future Cardiol., 15 53