The FDA has approved Attruby, a new oral treatment that helps reduce heart-related deaths and hospital visits among adults with transthyretin amyloid cardiomyopathy, or ATTR-CM. 

ATTR-CM is a rare but serious long-term heart condition that may be inherited or related to aging. It is caused by a buildup of weak and unstable transthyretin (TTR) protein that deposits to form amyloid fibrils. These amyloid clumps thicken and stiffen the heart muscles, which makes it harder for the heart to pump blood, leading to breathlessness, fatigue, fainting, abnormal heart rhythms, heart failure, and  a higher risk of an early death.

Attruby — the generic name for the drug is acoramidis — helps stabilize the weak TTR, reducing the buildup of the faulty protein and slowing disease progression. It is the first treatment to achieve nearly complete (90%) stabilization of TTR, making it better at improving heart health than other therapies. Studies have also shown that it provides benefits faster than the standard treatments.

A clinical trial that studied the safety and effectiveness of Attruby in 632 patients for 30 months showed that the drug led to better results in terms of survival, overall health, and daily functioning than a placebo. It significantly reduced deaths and hospital visits due to heart problems by 42% and lowered the risk of heart failure by about 50%.

Patients receiving Attruby showed mild side effects such as diarrhea and upper abdominal pain, both of which resolved easily. A few changes were seen in certain lab findings, but these values became normal when the treatment was stopped.

“Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalization as early as three months after initiation of therapy. With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition,” said Martha Grogan, MD, a cardiologist at the Mayo Clinic, in a statement. 

BridgeBio, the drug’s maker, will be offering a patient support program called ForgingBridges to U.S. patients prescribed Attruby and their families to help them get the therapy with insurance support, financial assistance, and a dedicated support team, according to their press release. 

SOURCES:

BridgeBio: “Attruby™ (acoramidis), a Near Complete TTR Stabilizer (≥90%), approved by FDA to Reduce Cardiovascular Death and Cardiovascular-related Hospitalization in ATTR-CM Patients.”

The New England Journal of Medicine: “Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.”

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