TOPLINE:

Among patients with transthyretin amyloid cardiomyopathy (ATTR-CM), early and continuous treatment with acoramidis through 54 months was associated with lower risks for all-cause mortality, cardiovascular mortality, and first cardiovascular hospitalization than delayed initiation of treatment, and no long-term safety concerns were observed.

METHODOLOGY:

• The phase 3 ATTRibute-CM trial involving patients with ATTR-CM showed promising outcomes with acoramidis through 30 months. In the ongoing open-label extension of this multicenter study, researchers reported the long-term efficacy and safety of the drug.
• A total of 389 patients with ATTR-CM who completed the 30-month trial were enrolled in the open-label extension between October 2021 and April 2025.
• Overall, 263 patients continued taking 800 mg oral acoramidis twice daily, and 126 switched from placebo to the same acoramidis regimen at month 30.
• Researchers assessed all-cause mortality, cardiovascular-related mortality, and first cardiovascular hospitalization over a median follow-up duration of 53.2 months.
• Additional analyses included determining the levels of N-terminal pro-B-type natriuretic peptide (NT-proBNP) and serum transthyretin (sTTR), 6-minute walk distance, scores indicating heart failure-related health status, and safety.

TAKEAWAY:

• Patients who continued acoramidis treatment through month 54 had a 45% lower risk for all-cause mortality and a 49% lower risk for cardiovascular-related mortality than those who switched from placebo to acoramidis (P < .001 for both).
• Continuous acoramidis treatment was associated with a 47% reduced risk for first cardiovascular hospitalization at month 54 (P < .001).
• Patients who continued acoramidis had stable NT-proBNP levels, sustained increases in sTTR levels (greater than in those who switched), and a slower decline in 6-minute walk distance and maintained heart failure-related health status at month 54. Those who switched from placebo to acoramidis had NT-proBNP levels that stabilized after switching and had improved 6-minute walk distance.
• No new safety concerns were reported during the extended follow-up through month 54. Treatment-emergent adverse events occurred in 94.3% of patients, with no difference between those who continued and those who switched to acoramidis.

IN PRACTICE:

“These findings demonstrate the long-term, sustained clinical benefits of acoramidis in ATTR-CM and emphasize the need for early diagnosis followed by prompt treatment in patients with ATTR-CM,” the researchers wrote.

SOURCE:

This study was led by Prem Soman, MD, PhD, of University of Pittsburgh Medical Center, Pittsburgh. It was published online on March 30, 2026, in JAMA Cardiology. The work was presented as an abstract at American College of Cardiology (ACC) Scientific Session 2026 in New Orleans.

LIMITATIONS:

Higher use of background therapies for heart failure at the entry to the open-label extension may have led to an underestimation of the efficacy of acoramidis, particularly among those who switched from placebo to acoramidis. Parameters related to disease progression, among other baseline characteristics, were imbalanced between groups and may have biased the results. The lack of a control group in the open-label study phase may have introduced uncertainty in interpreting the long-term safety outcomes.

DISCLOSURES:

The original and open-label extension studies received funding from BridgeBio. Several authors disclosed receiving personal fees, advisory or speaking fees, consultant fees, grants (including grants to authors’ institutions), and honoraria and serving as medical monitors and on speakers bureau with various companies both during the conduct of the study and outside the submitted work. One author disclosed that his institution served as a clinical trial site during the study and that he served as an unpaid consultant and advisor for BridgeBio. Some authors reported being employees and stockholders of BridgeBio, and one reported holding an issued patent for the company.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.