Abstract
Purpose
To provide an overview of the manifestation and diagnosis of transthyretin amyloid cardiomyopathy (ATTR-CM) and the treatment option acoramidis, including its pharmacology and clinical trial data.
Summary
ATTR-CM is a progressive, life-threatening cause of heart failure, resulting from destabilization of transthyretin (TTR), a tetrameric transport protein. TTR destabilization leads to monomers that misfold, aggregate, and deposit as amyloid fibrils in cardiac and other tissues. ATTR-CM is underdiagnosed, and the common cardiovascular manifestations associated with it are frequently misattributed to other types of heart disease, delaying diagnosis. ATTR-CM management has historically been largely supportive, but disease-modifying therapies, including TTR stabilizers and gene silencers, have become available in the past decade. Acoramidis, a next-generation oral TTR stabilizer, was approved in 2024 for the treatment of adults with wild-type or variant ATTR-CM based on the results of the phase 3 ATTRibute-CM trial. Acoramidis has been shown to achieve near-complete (≥90%) TTR stabilization, increase levels of serum TTR, reduce rates of cardiovascular-related mortality and hospitalization, and improve patient quality of life. Clinical pharmacists play a central role in the timely diagnosis and treatment of ATTR-CM, including facilitating access to therapies, monitoring safety concerns, and providing patient counseling.