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Published on: June 10, 2025

Effect of Acoramidis on Heart Failure-Related Health Status: A Secondary Analysis of the ATTRibute-CM Randomized

Charles F Sherrod1,2, Marianna Fontana3, Julian D Gillmore3

  • 1Healthcare Institute for Innovations in Quality, University of Missouri-Kansas City.

JAMA Cardiology
|July 29, 2026
PubMed

Insights

Acoramidis significantly improved patient-reported health status in transthyretin amyloid cardiomyopathy (ATTR-CM) by stabilizing transthyretin. This drug offers meaningful patient-centered benefits and modifies disease trajectory.

Area of Science:

  • Cardiology
  • Pharmacology
  • Clinical Trials

Background:

  • Transthyretin amyloid cardiomyopathy (ATTR-CM) significantly impacts patient health status.
  • Acoramidis stabilizes transthyretin, reducing mortality and hospitalizations in ATTR-CM.
  • Comprehensive data on acoramidis's effect on patient-reported health status is limited.

Purpose of the Study:

  • To evaluate acoramidis's impact on heart failure (HF)-related health status.
  • To assess changes using the Kansas City Cardiomyopathy Questionnaire Overall Summary score (KCCQ-OS).

Main Methods:

  • Phase 3, international, placebo-controlled ATTRibute-CM trial.
  • 30-month treatment with acoramidis (800 mg twice daily) or placebo.
  • Analysis of KCCQ-OS using mixed-effects model for repeated measures.

Main Results:

  • Acoramidis demonstrated a statistically significant and clinically meaningful improvement in KCCQ-OS (LSM difference, 9.9; P<.001).
  • At 30 months, acoramidis recipients were more likely to be 'alive and not worse' (47% vs 30%), 'alive and well' (46% vs 31%), and 'alive and better' (25% vs 14%).
  • Number needed to treat (NNT) ranged from 6 to 9 for these outcomes.

Conclusions:

  • Acoramidis significantly attenuated the decline in HF-related health status compared to placebo.
  • Results suggest meaningful patient-centered benefits and clinically relevant disease modification.
  • Acoramidis offers a promising therapeutic option for improving quality of life in ATTR-CM patients.
Abstract

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