New data shows that long-term treatment with acoramidis is associated with lower mortality and fewer hospitalizations in people with transthyretin amyloid cardiomyopathy (ATTR-CM), according to a study published in JAMA Cardiology.
In an extension of the ATTRibute-CM clinical trial, researchers followed 389 participants for up to 54 months. Patients who received acoramidis for the entire study period had a 45% lower risk of death overall and a 49% lower risk of death due to heart disease compared with those who started the study on a placebo and switched to acoramidis later on. They also had a 47% lower risk of cardiovascular hospitalization.
In ATTR-CM, misfolded transthyretin (TTR) protein builds up in the heart, leading to heart failure over time. The oral therapy acoramidis works by stabilizing this protein and preventing harmful deposits from forming.
In the study, acoramidis increased levels of serum transthyretin (sTTR), which suggests the protein is being stabilized. Patients who started treatment later also showed increases in sTTR, though to a lesser extent, possibly reflecting more advanced disease at treatment initiation.
Read more about ATTR-CM therapies
Measures of quality of life remained stable in patients treated continuously with acoramidis. Those who started treatment later also showed gradual improvements, although benefits were greater when treatment was initiated earlier. Levels of NT-proBNP (N-terminal pro–B-type natriuretic peptide), a marker of heart failure severity, were stable with continuous treatment.
Patients also experienced a slower decline in physical function, measured by how far they could walk in six minutes — a common measure used to assess daily activity levels. No new safety concerns were identified during long-term follow-up.
Benefits became more pronounced with longer treatment, particularly by month 42, and were sustained through month 54. Patients who first took a placebo for 30 months had smaller gains in stabilization and functional improvement than those who took acoramidis for the entire study.
“Taken together, these findings confirm the importance of prompt diagnosis and treatment, while recognizing that patients presenting at an advanced stage may still benefit from therapy,” the authors concluded.
The study has some limitations. Because all participants eventually received acoramidis, there was no long-term placebo comparison, and differences between groups at the start of the extension phase, as well as the use of other treatments, may have influenced the results.
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