- Question
- Does acoramidis, a high-affinity transthyretin (TTR) stabilizer, improve mortality, morbidity and functional outcomes in patients with transthyretin amyloid cardiomyopathy?
- Design
- Phase 3, randomized 2:1, double-blind, placebo-controlled trial with 632 patients randomized and 30 months of treatment. Efficacy was assessed in patients with an estimated glomerular filtration rate of at least 30 ml/min/1.73 m2.
- Population
- Patients with symptomatic transthyretin amyloid cardiomyopathy.
- Intervention
- Acoramidis hydrochloride 800 mg twice daily for 30 months.
- Control
- Matching placebo twice daily for 30 months.
- Primary outcome
- Four-step hierarchical analysis comprising death from any cause, cardiovascular-related hospitalization, change from baseline in NT-proBNP, and change from baseline in 6-minute walk distance, compared using the Finkelstein-Schoenfeld method.
- Results
- The primary analysis favored acoramidis (P<0.001), with a win ratio of 1.8 (95% CI, 1.4 to 2.2); 63.7% of pairwise comparisons favored acoramidis and 35.9% favored placebo. Death from any cause and cardiovascular-related hospitalization contributed more than half of the wins and losses (58% of comparisons), and NT-proBNP had the highest win-to-loss ratio (23.3% vs. 7.0%).
- Safety
- The overall incidence of adverse events was similar with acoramidis and placebo (98.1% and 97.6%); serious adverse events occurred in 54.6% and 64.9%, respectively.
- Conclusion
- In transthyretin amyloid cardiomyopathy, acoramidis produced a significantly better hierarchical composite of mortality, morbidity and function than placebo, with similar adverse events in both groups.
- Why it matters
- It provided evidence that stabilizing transthyretin with a high-affinity oral drug can modify the clinical course of this disease, broadening the available disease-specific treatment options.
Card generated automatically with AI from the abstract of the original publication and the trial registry; every figure was checked against those sources. It may contain errors: verify the data at the source before using it for clinical decisions.
Generated on October 14, 2026