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News From the Food and Drug Administration
June 18, 2019

Drugs for Heart Disease With Rare Cause

JAMA. 2019;321(23):2274. doi:10.1001/jama.2019.7668

The FDA recently approved 2 drugs for adults with cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR), a rare and life-threatening disease. The oral medications, tafamidis meglumine and tafamidis, are the first to receive agency approval for this indication.

Transthyretin-mediated amyloidosis develops when misfolded transthyretin proteins accumulate abnormally and form amyloid fibrils—a process that occurs most frequently in the heart and peripheral nervous system. Buildup of misfolded transthyretin proteins in the myocardium can result in fatigue, shortness of breath, abnormal heart rhythms, and heart failure. Cardiac events are the most common cause of death in patients with ATTR.