Navigating Therapeutic Pathways and Transforming Care in ATTR-CM - Episode 9
Efficacy and Safety of Transthyretin Stabilizers in ATTR-CM
The panel discusses the role of transthyretin (TTR) stabilizers in the management of ATTR-CM, focusing on tafamidis and acoramidis. They explain that both agents work by stabilizing the TTR tetramer to prevent dissociation and subsequent amyloid fibril formation. Tafamidis is highlighted as the first approved stabilizer with demonstrated benefit in reducing morbidity and mortality in ATTR-CM. Clinicians note that many patients experience stable disease over time with tafamidis, and its favorable tolerability profile, with minimal need for laboratory monitoring, is especially helpful for older adults seeking consistent, manageable treatment. The discussion then shifts to acoramidis, which is designed to strongly stabilize the TTR protein. The panel describes clinical impressions that acoramidis has shown promising results in trials and represents an important emerging option. They emphasize that having multiple stabilizers broadens therapeutic choice and may help align treatment selection with patient needs and disease characteristics, reinforcing the importance of earlier diagnosis and appropriate therapy initiation.
The panel discusses the role of transthyretin (TTR) stabilizers in the management of ATTR-CM, focusing on tafamidis and acoramidis. They explain that both agents work by stabilizing the TTR tetramer to prevent dissociation and subsequent amyloid fibril formation. Tafamidis is highlighted as the first approved stabilizer with demonstrated benefit in reducing morbidity and mortality in ATTR-CM. Clinicians note that many patients experience stable disease over time with tafamidis, and its favorable tolerability profile, with minimal need for laboratory monitoring, is especially helpful for older adults seeking consistent, manageable treatment.
The discussion then shifts to acoramidis, which is designed to strongly stabilize the TTR protein. The panel describes clinical impressions that acoramidis has shown promising results in trials and represents an important emerging option. They emphasize that having multiple stabilizers broadens therapeutic choice and may help align treatment selection with patient needs and disease characteristics, reinforcing the importance of earlier diagnosis and appropriate therapy initiation.
