A research review published in Cardiology and Therapy highlights the major progress that’s been made in recent years when it comes to treating transthyretin amyloid cardiomyopathy (ATTR-CM). Once treated only with symptom management, ATTR-CM now has several therapies that target the disease itself, and more are being developed.
Until recently, treatment plans for ATTR-CM focused on controlling symptoms, mostly by using diuretics to reduce fluid buildup. But in the past few years, therapies that address the underlying cause of the disease have received FDA approval and are making a big difference in patients’ lives.
One such medication is tafamidis, a daily pill that stabilizes the faulty TTR protein, which misfolds and builds up in the heart. In a clinical trial, tafamidis was associated with a significant reduction in both death and cardiovascular-related hospitalizations over a 30-month period.
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In 2024, the FDA approved a newer drug called acoramidis, which works similarly to tafamidis but may be even more effective at preventing disease progression.
Another major breakthrough comes from gene-silencing therapies, like patisiran and vutrisiran; the latter was approved by the FDA for ATTR-CM in March of this year. These medications reduce the body’s production of the faulty TTR protein. Early data show that these drugs help patients maintain better heart function and reduce hospitalizations.
Researchers are also exploring emerging, cutting-edge treatments like gene editing and antibody therapies that may one day remove amyloid deposits or stop the disease entirely.
With the current pace of development, the study’s authors say they expect the landscape for ATTR-CM treatments to look different even within the next five years. As newer therapies become available, they call for more personalized treatment plans that take into account individual symptoms, biomarkers and patient preferences.
“ATTR-CM management is no longer a one-size-fits-all approach,” the authors conclude. “The next few years will define not just which therapies are available, but how they are used.”
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