Acoramidis is an orally taken small‑molecule medicine developed to treat transthyretin amyloid cardiomyopathy (ATTR‑CM), a progressive form of heart disease caused by misfolding and deposition of transthyretin protein in cardiac tissue; it works by strongly stabilizing the transthyretin tetramer, preventing it from breaking down and forming harmful amyloid deposits. Originally identified as a compound called AG10 in academic research, its development continued through clinical programs that showed significant reductions in cardiovascular death and hospitalizations in patients with both wild‑type and hereditary forms of ATTR‑CM, leading to its first regulatory approval in the United States in November 2024 and later approvals in other regions.
