The European Commission approved acoramidis to treat adults with wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM) in the European Union, Bayer announced recently. Bayer will market acoramidis in the EU under the trade name Beyonttra beginning in the first half of the year.
The U.S. Food and Drug Administration approved acoramidis in November for use in the U.S., where BridgeBio Pharma Inc. markets it under the brand name Attruby.
What is ATTR-CM?
Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is a rare progressive disease of the heart muscle that leads to congestive heart failure. It occurs when the transthyretin protein produced by the liver is unstable. Symptoms include fatigue; shortness of breath; irregular heart rate or palpitations; swelling of the legs, ankles and stomach; brain fog; wheezing; and dizziness. It often goes underdiagnosed because of a lack of awareness and knowledge of the disease. There is currently no cure for ATTR-CM.
Acoramidis is an oral medication that slows progression of the disease by stabilizing the transthyretin (TTR) protein, reducing amyloid buildup in the heart.
Read more about ATTR-CM therapies
The European Commission decision followed results of the Phase III ATTRibute-CM trial, in which the drug demonstrated significant benefits, including a reduction in cardiovascular-related hospitalizations and mortality.
“Ongoing advancements in treatment are important because they have the potential to significantly improve outcomes in this fatal disease,” Marianna Fontana, professor of cardiology at University College London and honorary consultant cardiologist at the National Amyloidosis Centre in the U.K., said in a statement. Acoramidis “was specifically developed to decelerate symptom progression and improve outcomes in patients with ATTR-CM by providing near-complete stabilization of transthyretin.”
ATTR-CM is caused by the misfolding of TTR, which leads to amyloid deposits in heart tissue, impairing its function. The disease is progressive and can lead to heart failure, arrhythmias and reduced life expectancy.
In the clinical study, acoramidis showed effectiveness as early as three months after initiation of treatment. The trial also found that patients who switched from a placebo to acoramidis at 30 months experienced measurable improvements, reinforcing its long-term potential. An open-label extension study further demonstrated a 34% reduction in mortality risk by 42 months, highlighting its role in prolonging survival.
ATTR-CM is often underdiagnosed, and untreated cases can rapidly progress to severe heart failure. Acoramidis represents an additional option for physicians treating the condition, complementing existing therapies, Bayer said. Its long-term effect on patient outcomes continues to be studied.
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