While there have been significant therapeutic advancements in transthyretin amyloid cardiomyopathy (ATTR-CM) in recent years, none cure the disease.
Many patients will find themselves sifting through studies on the latest developments in treatment, and see mentions of promising new experimental therapies. Deciding to pursue one of these therapies should be a decision made between you and your doctor, with full awareness of both the FDA-approved therapies currently available and the drawbacks of experimental therapies.
The changing landscape of ATTR-CM therapies
Just a few years ago, no therapies specifically for ATTR-CM existed. Recent drug approvals have shaken the ATTR-CM treatment landscape in a dramatic way.
In 2019, tafamidis (marketed as Vyndamax or Vyndaqel) was granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of ATTR-CM. Tafamidis stabilizes the TTR tetramer, thereby reducing the formation of amyloid fibrils. More recently, two more drugs gained approval as ATTR-CM treatments. Acoramidis (otherwise known as Attruby), another transthyretin stabilizer, received U.S. FDA approval in 2024. This was followed in 2025 by vutrisiran (marketed as Amvuttra), an RNA interference therapeutic that silences TTR production.
Thanks to these new therapies, patients with ATTR-CM are living longer and with better quality of life. But science hasn’t stopped working towards more treatments.
Experimental therapies for ATTR-CM
Several experimental therapies are currently in the pipeline for ATTR-CM.
NTLA-2001 uses a therapeutic technique known as gene editing, which allows doctors to directly edit the gene responsible for causing symptoms of the disease. NTLA-2001 is delivered in a single dose. Research has already indicated that it can result in long-lasting transthyretin reduction.
Another experimental therapy is PRX004. PRX004 is known as a monoclonal antibody, which is a laboratory-produced antibody designed to target harmful substances in the body, just like normal antibodies produced by the immune system do. In the case of the PRX004, its mechanism of action is to target and clear away misfolded transthyretin proteins that are the underlying cause of ATTR-CM disease activity.
Read more about ATTR-CM testing and diagnosis
Accessing experimental therapies
For your own safety, the U.S. Food and Drug Administration does not allow experimental therapies to be made broadly available to the public. Generally, the only way you can access an experimental therapy is if you enroll in a clinical study testing the drug; however, you might receive the placebo instead of the experimental therapy.
There is also legislation called “The Right to Try Act,” which allows patients to try experimental therapies when they are faced with a life-threatening illness.
Patients exploring experimental therapies need to manage their expectations: these therapies are not fully developed, and can fail or even cause harm. FDA-approved therapies have undergone rigorous trials demonstrating they provide clear clinical benefits without intolerable side effects. Experimental therapies, on the other hand, may not be effective, or may cause significant side effects. FDA approval is often a drawn-out process that takes years, so it will take time for any experimental therapy’s value to be understood and for it to become available to patients.
Despite these cautions, there are stories of individuals who have taken experimental therapies and have seen their lives improve. If you’re interested in these therapies, talking with your care team is the best place to start.
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