Ann Pharmacother. 2025 Sep 21:10600280251368386. doi: 10.1177/10600280251368386. Online ahead of print.
ABSTRACT
OBJECTIVE: The objective of the study was to review acoramidis, a new transthyretin stabilizer, for treatment of transthyretin amyloid cardiomyopathy by means of pharmacology, efficacy, and safety.
DATA SOURCES: An Embase, PubMed, and ClinicalTrials.gov search was conducted using the keywords acoramidis, Attruby, and AG10.
STUDY SELECTION AND DATA EXTRACTION: We included full-text, English-language studies that evaluated the pharmacology, efficacy, and safety of acoramidis in transthyretin amyloid cardiomyopathy.
DATA SYNTHESIS: Acoramidis slows or halts the accumulation of amyloid deposits in the heart by binding to the thyroxine-binding sites on the tetrameric transthyretin (TTR) protein preventing the TTR tetramer from dissociating into monomers. Acoramidis did not achieve statistical significance in terms of mortality reduction, but it outperformed placebo with respect to death from any cause, cardiovascular-related hospitalization, change in N-terminal pro-B-type natriuretic peptide, and change in 6-minute walk distance. In addition, by the end of the phase II clinical trial all acoramidis-treated patients achieved normal serum TTR concentrations. The overall incidence of subjects who experienced any treatment-emergent adverse events was similar between the acoramidis and placebo groups.Relevance to patient care and clinical practice in comparison to existing drugs:Acoramidis is the second TTR stabilizer approved for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Each medication to manage ATTR-CM similarly decreases the combined endpoints of all-cause mortality and progression of heart failure symptoms, when adjusted for risk factors. However, its place in therapy remains unclear among the treatment options for the management of ATTR-CM due to the lack of head-to-head trials.
CONCLUSIONS: Acoramidis is an effective and safe medication for the treatment of transthyretin amyloidosis cardiomyopathy.
PMID:40975800 | DOI:10.1177/10600280251368386
