Acoramidis Significantly Improves Quality of Life in Amyloid Cardiomyopathy: JAMA
USA: A secondary analysis of the ATTRibute-CM randomized clinical trial has shown that, beyond improving survival and reducing hospitalizations, acoramidis significantly slowed the decline in heart failure-related health status compared with placebo in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The findings highlight meaningful patient-centered benefits and suggest that acoramidis may favorably alter the disease trajectory in this progressive cardiac condition.
The study, published in JAMA Cardiology, was conducted by Charles F. Sherrod IV, MD, MSc, Healthcare Institute for Innovations in Quality, University of Missouri–Kansas City, and colleagues.
ATTR-CM is a progressive disease caused by the deposition of misfolded transthyretin protein in the heart, leading to worsening heart failure symptoms, reduced quality of life, and increased mortality. While acoramidis has previously been shown to stabilize transthyretin protein and reduce deaths and cardiovascular-related hospitalizations, its impact on patient-reported health status had not been fully evaluated.
To address this, researchers performed a secondary analysis of the phase 3, multicenter, international, placebo-controlled ATTRibute-CM trial, conducted between April 2019 and May 2023. The trial enrolled adults with ATTR-CM, with data analyzed from July to November 2023. Participants were randomly assigned to receive either acoramidis hydrochloride 800 mg twice daily or placebo for 30 months.
The analysis included 611 patients in the modified intention-to-treat population, comprising 409 patients in the acoramidis group and 202 in the placebo group. The average age of participants was 77.2 years, and 9.2% were women. Heart failure-related health status was assessed using the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score.
The trial revealed the following findings:
- At 30 months, acoramidis significantly slowed the decline in heart failure-related health status compared with placebo, with a clinically meaningful 9.9-point improvement in Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) scores.
- Nearly half of patients receiving acoramidis (47%) were "alive and not worse"—surviving without a decline of 5 or more points in KCCQ-OS—compared with 30% of patients receiving placebo.
- A higher proportion of patients treated with acoramidis were "alive and well" (46% vs 31% with placebo), indicating survival with preserved health status.
- More patients in the acoramidis group were "alive and better" (25% vs 14% with placebo), reflecting an improvement of more than 5 points in KCCQ-OS from baseline.
The researchers acknowledged several limitations, including the addition of the KCCQ assessment through a protocol amendment, resulting in missing month 3 data for some participants. The relatively low representation of women and patients with variant ATTR-CM may also limit the generalizability of the findings, while earlier diagnosis in current clinical practice could influence outcomes.
The authors concluded that, in addition to improving survival and reducing cardiovascular hospitalizations, acoramidis meaningfully preserves patient-reported heart failure-related health status. These sustained benefits were consistent across prespecified subgroups, supporting its role in favorably modifying the disease course of ATTR-CM.
Reference:
Sherrod CF, Fontana M, Gillmore JD, et al. Effect of Acoramidis on Heart Failure–Related Health Status: A Secondary Analysis of the ATTRibute-CM Randomized Clinical Trial. JAMA Cardiol. Published online July 29, 2026. doi:10.1001/jamacardio.2026.2413
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