Current available treatment options targeting TTR tetramer stabilization

Abstract Transthyretin (TTR) stabilization is a well-established method for the treatment of transthyretin amyloidosis, particularly involving the associated diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM). Four different oral pharmacologic TTR stabilizers have been used or proposed for treatment of this condition: diflunisal, tafamidis, tolcapone, and acoramidis. Two of them (acoramidis and tafamidis) have received international approval for use in patients with ATTR-CM, based on double-blinded, placebo-controlled clinical trials. To date, there have not been any head-to-head studies comparing the efficacy of these approved TTR stabilizers. This article describes the background and data supporting the use of tafamidis and acoramidis in people with ATTR-CM, including reports of blinded in vitro comparison, and surrogate markers for TTR tetramer stabilization, such as serum transthyretin levels.