News

Cardiac amyloidosis caused by transthyretin is a rare but severe cardiomyopathy caused by aberrant accumulation of misfolded transthyretin protein (TTR) in the myocardium. If untreated, transthyretin amyloid cardiomyopathy (ATTR-CM) is characterized by progressive worsening heart failure with a median survival of less than 4 years.

Current approved TTR-specific therapies either block the formation of mutated TTR protein by mRNA silencing (vutrisiran) or stabilize the TTR tetramer to prevent misfolded transthyretin protein (tafamidis). The next generation TTR stabilizer acoramidis has recently been approved for the treatment of ATTR-CM due to its success in the ATTRibute-CM trial. The current drug pipeline includes promising new treatment approaches for ATTR-CM. The most advanced clinical projects will be discussed in more detail.