3. Inaugural medication for transthyretin amyloid cardiomyopathy
ATTR-CM is a cardiovascular disorder where amyloid protein fibrils deposit in the walls of the heart’s left ventricle and stiffen. The heart condition is underdiagnosed and potentially fatal, according to the Cleveland Clinic.
A new agent could prevent misfiling of deposited protein and has been shown to significantly reduce risk of death.
“The medication binds the protein and stabilizes so it doesn’t break apart in the heart,” Dr. Mazen Hanna, a staff member in cardiovascular medicine at the Cleveland Clinic, said. “It’s a huge breakthrough in the field for a very underdiagnosed disease.”
The medication received fast-track and breakthrough designations in 2017 and 2018 and the FDA approved tafamidis medication in 2019 to treat ATTR-CM.