Acoramidis: A New Transthyretin Stabilizer for Transthyretin Amyloid Cardiomyopathy.

Authors

Taylor Clark, Rachel Lucas, Azadeh Nasuhidehnavi, Nathan Sauers, Kelly Bach

Published in

The Annals of pharmacotherapy. Pages 10600280251368386. Sep 21, 2025. Epub Sep 21, 2025.

Abstract

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.
An Embase, PubMed, and ClinicalTrials.gov search was conducted using the keywords acoramidis, Attruby, and AG10.
We included full-text, English-language studies that evaluated the pharmacology, efficacy, and safety of acoramidis in transthyretin amyloid cardiomyopathy.
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.
Acoramidis is an effective and safe medication for the treatment of transthyretin amyloidosis cardiomyopathy.

PMID:
40975800
Bibliographic data and abstract were imported from PubMed on 21 Sep 2025.

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