Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, often fatal condition driven by misfolded transthyretin (TTR) protein deposits in the heart muscle. Acoramidis (Attruby; BridgeBio Pharma), a near-complete oral TTR stabilizer approved for ATTR-CM, demonstrated significant clinical benefits through 30 months in the phase 3 ATTRibute-CM trial.1 Now, an open-label extension (OLE) of that trial reports that those benefits are not only sustained but amplified with continued early treatment through 54 months of follow-up.
Published in JAMA Cardiology, the OLE enrolled 389 patients with ATTR-CM who completed the 30-month ATTRibute-CM trial between October 2021 and April 2025. Patients who had received acoramidis during the trial continued treatment (continuous acoramidis group), while those who had received placebo were switched to acoramidis at OLE entry (delayed initiation group).