No. 3 Medical Innovation for 2020: Inaugural Medication 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.”