Keyur B. Shah, MD, Virginia Commonwealth University Health, discusses the approval of acoramidis for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
ATTR-CM is a rare amyloidosis caused by aging or genetic mutations. This results in the misfolding of TTR protein and accumulation of amyloid fibers in the cardiac myocardium. TTR fibrils build up in tissues, such as the peripheral nerves and heart, as well as the gastrointestinal tract, eyes, kidneys, central nervous system, thyroid, and bone marrow. This accumulation causes progressive dysfunction of these tissues. The disease eventually leads to heart failure.
Acoramidis is a near-complete stabilizer of TTR approved by the U.S. Food and Drug Administration for the treatment of adults with ATTR-CM. The approval follows positive results from the phase 3 ATTRibute-CM study where acoramidis demonstrated significant benefit on mortality, hospitalizations, and quality of life. Additionally, the treatment was generally well-tolerated.
To learn more about ATTR-CM and other rare heart conditions, visit https://checkrare.com/diseases/heart-diseases/
