Am J Health Syst Pharm. 2026 Jun 22:zxag181. doi: 10.1093/ajhp/zxag181. Online ahead of print.
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.
CONCLUSION: It is important for pharmacists to become familiar with the clinical manifestations of ATTR-CM and available treatment options. This review provides comprehensive information on ATTR-CM and the treatment landscape, focusing on the treatment option acoramidis.
PMID:42329158 | DOI:10.1093/ajhp/zxag181