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

Tafamidis is first FDA-approved drug to treat potentially fatal disease

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.

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