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◆ Journal of cardiovascular development and disease2026-07-26

Real-World Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy: A Pharmacovigilance Study.

Gregory W Chai, Quynh Phi, Diego Herman Delgado

原始摘要(英文原文)· Original abstract
Acoramidis is a novel transthyretin stabilizer recently approved for the treatment of transthyretin amyloid cardiomyopathy. Although clinical trials showed that the drug was generally safe, comprehensive real-world safety data after its approval remain limited. This study characterizes the real-world safety profile of acoramidis using the FDA Adverse Event Reporting System (FAERS) database. The database was queried for adverse events associated with acoramidis as the primary drug suspect from 1 January 2022 through 31 December 2025. Following deduplication and removal of confounding adverse events that could have stemmed from the underlying amyloidosis, a dual-statistical framework was applied. Frequentist disproportionality analysis was followed by Bayesian shrinkage using a Bayesian Confidence Propagation Neural Network to isolate statistically robust safety signals. Frequentist disproportionality analysis of 290 unique cases identified 25 safety signals. Subsequent Bayesian refinement isolated nine signals that met both frequentist and Bayesian criteria. The dual Bayesian and frequentist approaches identified signals of renal stress and gastrointestinal distress. Neuromuscular and systemic signals such as fatigue and dizziness met frequentist thresholds but did not meet the Bayesian threshold. This study's findings reflect the previously reported gastrointestinal distress and disproportionate laboratory shifts in renal function. This study's real-world post-marketing surveillance of acoramidis reinforces the safety data that was established through the clinical trial, ATTRibute-CM.
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Real-World Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy: A Pharmacovigilance Study. — 科研速览 Science Skim