Patients with transthyretin amyloid cardiomyopathy (ATTR-CM) are beginning to see real progress in treatment outcomes thanks to disease-modifying therapies such as tafamidis and acoramidis, according to a review published recently in Current Heart Failure Reports.
As this review explained, these medications are helping people live longer and maintain their quality of life by slowing progression of disease and reducing heart-related hospital visits.
“CA [cardiac amyloidosis] has been long considered a rare condition characterized by poor prognosis,” explained the review’s authors. “However, many advances in therapeutic strategies have transformed the landscape of this disease. The standard of care for ATTR-CA actually includes stabilizing agents such as tafamidis, which is the only drug approved for this condition.”
In 2019 tafamidis became the first drug approved by the FDA for ATTR-CM, after being initially used to treat ATTR with polyneuropathy. In a large clinical trial known as ATTR-ACT, 441 patients were studied over 30 months. Those who received tafamidis saw a 30% drop in all-cause mortality and a significant reduction in cardiovascular hospitalizations.
In addition, patients experienced less decline in their ability to walk and reported better overall well-being. However, it took about 18 months for the survival benefits to clearly show. Patients in more advanced stages of the disease — specifically New York Heart Association class III — saw fewer benefits at first, though long-term follow-up revealed some improvement even for them.
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Acoramidis, approved in 2024, has become another promising option. In the ATTRibute-CM trial involving 632 patients, acoramidis users showed a clear advantage in a combination of outcomes, including reduced mortality and fewer heart-related hospitalizations. Patients also maintained better physical function and had lower levels of a marker tied to heart stress. Though acoramidis did not improve walking ability in the first 12 months, patients reported feeling better, and lab results confirmed the drug was working to stabilize the disease.
Both medications appear most effective in people with earlier stages of ATTR-CM. They work by stabilizing the transthyretin protein that causes the damaging buildup in the heart. Tafamidis has been shown to slow both systolic and diastolic heart decline, while acoramidis improves heart biomarkers and patient-reported outcomes.
Despite these advances, cost remains a challenge — tafamidis, for example, carries a list price around $250,000 per year, far beyond traditional cost-effectiveness thresholds. Many experts argue the price would need to drop by more than 90% to be considered affordable at scale.
Still, the results of these clinical trials bring hope. For patients with ATTR-CM, these treatments mark a new era of managing the disease — offering not just symptom relief but the potential to live longer with better health.
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