The U.S. Food and Drug Administration (FDA) has granted fast-track status to a new drug that may reverse heart damage in transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This condition occurs when a protein called transthyretin (TTR) misfolds and builds up in the heart, causing damage. The drug, currently known as ALXN2220, is a depleter monoclonal antibody that helps the immune system clear harmful amyloid deposits.

In early trials, ALXN2220 significantly reduced markers of amyloid buildup in the heart in participants with wild-type or variant ATTR-CM and heart failure. Researchers used imaging tests to track changes in amyloid levels and found noticeable improvements over 12 months. These improvements suggest the drug may help reverse heart damage. ALXN2220 also improved heart health indicators, and no major side effects were reported during the study. The drug is now in phase 3 trials to see if it can help people live longer and reduce heart-related complications.

The FDA grants fast-track status to drugs for serious conditions that currently have limited treatment options. It speeds up the approval process by allowing closer collaboration with the FDA.

New Hope for Heart Repair

In ATTR-CM, amyloid deposits make the heart stiff, potentially leading to heart failure. Existing treatments like tafamidis (Vyndaqel) slow the disease but can’t remove the proteins already deposited in the heart. ALXN2220’s ability to clear these deposits may reverse heart damage, offering a potential new treatment option for people with this condition.

The first trial involved only 40 participants, so more testing is needed to determine whether the drug is effective on a larger scale. In the meantime, if you’re living with amyloidosis and concerned about cardiomyopathy, work with your cardiologist or other health care provider to ensure you’re receiving the best possible treatment.

Learn more about how amyloidosis can affect the heart, skin, kidneys, and more.

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Do you have heart-related symptoms from amyloidosis? Would you consider trying a new drug that may reverse heart damage? What tips would you share with others? Post your thoughts in the comments, or start a conversation on your Activities page.