Acoramidis

ATC Code C01EB25

Acoramidis

Definition

A potent, highly selective, orally bioavailable transthyretin (TTR) stabilizer with potential disease-modifying activity. Upon oral administration, acoramidis binds to and stabilizes transthyretin (TTR), thereby preventing tetramer dissociation into monomers. This prevents misfolding of the TTR protein and inhibits the formation of TTR amyloid fibrils and the subsequent deposition of these insoluble protein clusters in the heart and peripheral nerves. TTR is a transport protein for thyroxine and retinol and is secreted by the liver into the blood. The accumulation of TTR amyloid fibrils may result in thickening and stiffening of the ventricular wall, leading to heart failure.

Also known as ACORAMIDIS, AG 10, AG-10, AG10, ALXN 2060, ALXN-2060, ALXN2060, Transthyretin Stabilizer AG10, TTR Stabilizer AG10 — per NCIT

Also identified as

Indications

Disease Relation Source
Cardiomyopathy may treat MEDRT · Public domain (U.S. Government work)

Organ Effects

Organ Side effect / interaction Source
Cardiovascular system may treat a disease of this organ DERIVED · CC BY 4.0
Heart may treat a disease of this organ DERIVED · CC BY 4.0
Musculature may treat a disease of this organ DERIVED · CC BY 4.0
Musculoskeletal system may treat a disease of this organ DERIVED · CC BY 4.0
Myocardium may treat a disease of this organ DERIVED · CC BY 4.0

5 entries above are marked DERIVED: Atlas Médico inferred them by combining two sourced claims, and no source asserts these links directly. Treat them as a navigation aid, not as a clinical statement.