No. 3 Medical Innovation for 2020: Inaugural Medication for Transthyretin Amyloid Cardiomyopathy

Until 2019, the sole treatment options for people with transthyretin amyloid cardiomyopathy were symptom management and, in some patients, heart transplant. That changed in May, when the FDA approved the small-molecule compound tafamidis for the treatment of this progressive and potentially fatal disease.

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According to results of the phase 3 ATTR-ACT study, tafamidis significantly reduced all-cause mortality at 30 months relative to placebo (30 percent vs. 43 percent) and resulted in fewer cardiovascular-related hospitalizations.

FDA approval of this breakthrough drug may be the first step in turning transthyretin amyloid cardiomyopathy from a life-threatening condition into a manageable chronic disease.

In this video, learn more about why tafamidis is one of Cleveland Clinic’s Top 10 Medical Innovations for 2020.

Inaugural Treatment for Transthyretin Amyloid Cardiomyopathy

“Heart failure and atrial fibrillation are two of the most common cardiac diagnoses of the elderly population. Though previously underdiagnosed, it is now clear that a condition called amyloidosis is an important cause of both of these conditions. When amyloidosis affects the heart, it is known as amyloid cardiomyopathy, a progressive and potentially fatal disease that results from destabilized transthyretin, a transport protein. It becomes misfolded and forms amyloid fibrils that deposit into the walls of the heart, stiffening the muscle, eventually leading to heart failure.

Until now, treatment for the condition has been limited to transplant. In May, an approval brought hope to these suffering individuals. Two drug formulations of the compound known as tafamidis have been dubbed the first ever treatment for the two subtypes of the condition, hereditary and wild type. Phase three clinical trial results showed a 30 percent reduced risk of death in patients receiving this breakthrough therapy.

The approval of a drug for these conditions effectively highlights the importance of providing a treatment option where there once was none.”