Acoramidis for symptomatic transthyretin amyloid cardiomyopathy

Acoramidis is in clinical development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is an underdiagnosed, progressively debilitating, rare disease associated with high mortality. ATTR-CM occurs when a protein called transthyretin (TTR) becomes unstable and aggregates, forming amyloids, and deposits primarily in the heart, as well as other organs, known as transthyretin amyloidosis. This damages the structure and function of the organs and causes serious disease which can be fatal. It causes the thickening and stiffening of the heart tissues. Symptoms include shortness of breath, palpitations and abnormal heart rhythms, ankle swelling, fatigue, fainting and chest pain. Current treatment options for ATTR-CM are limited and mainly focus on symptom management and supportive care. However, heart and/or liver transplantation may be an option for some patients if appropriate, although rare.