What the trial was testing
The ATTRibute-CM enrolled 632 patients with amyloidosis. The study was sponsored by Eidos Therapeutics, a BridgeBio company and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Acoramidis reduced death or first hospitalization risk by 31% in the most common form of this rare heart disease.
JAMA cardiology · 2026 · NCT03860935
These findings — that death or first heart hospitalization risk cut by about 1 in 3 at 30 months — were published in the JAMA cardiology and represent the headline result of the study.
Researchers tracked outcomes across 632 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This is the first treatment shown to meaningfully extend survival and reduce hospitalizations in amyloid cardiomyopathy patients. If you or a loved one has been diagnosed with this condition, ask your doctor whether this pill might be right for you. The drug is FDA-approved and available now.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open amyloidosis trials
Prospective Study of Teclistamab in the Treatment of Systemic AL Amyloidosis
This study aims to evaluate the use of teclistamab in systemic AL amyloidosis and answer whether teclistamab can improve the rate of complete hematological response. This is a single-arm, multi-center, prospective study. Participants will receive the single drug teclistamab, which the investigator deems the best choice.
Thailand ATTR-CM Registry
The investigators of this registry aim to study the natural history, clinical presentation, characteristics, and imaging findings of patients diagnosed with ATTR amyloidosis in Thailand longitudinally. This will enable the investigators to study this disease in depth. With a better understanding, the investigators can aim to develop early screening programs for at-risk patients, raising awareness among non-cardiologists. As amyloid-specific therapies have now become available in Thailand, the findings of this registry can be helpful for epidemiological studies in Thailand.