Eplontersen for treating transthyretin amyloid cardiomyopathy

Eplontersen is in clinical development for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM occurs due to a build-up of abnormal proteins in the heart. These protein deposits can cause thickening and stiffening of the heart muscle, leading to heart failure. There are two types of ATTR, namely the hereditary and wild-type forms. Wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) is an underrecognized cause of heart failure due to misfolded wild-type transthyretin (TTRwt) myocardial deposition. Hereditary ATTR amyloidosis is caused by a mutation in the gene for TTR, inherited from one parent. The disease therefore runs in families, though the timing, development and severity of the disease can vary greatly. Wild-type ATTR-CM is the more common form of the condition. It is primarily seen in older men and often in conjunction with other age-related cardiac diseases. Current treatment options act by slowing the progress of the disease but no cure is available. There is a need for more treatments with increased effectiveness because the current options are limited.