Acoramidis Triumph: Safe & Effective in Transthyretin Amyloid Cardiomyopathy

The heart, which pumps over 100,000 times per day, is a vital muscle that transports oxygen and nutrients to our organs for us to operate normally. Unfortunately, heart failure affects around 6.2 million people in the United States and a staggering 64 million people worldwide.

One in every five older people with heart failure and abnormally thickened hearts has transthyretin amyloid cardiomyopathy, or ATTR-CM, which is underdiagnosed, rapidly progressive, and deadly. The condition, which can be genetic or arise spontaneously, is characterized by the buildup of misfolded transthyretin (TTR) proteins in the heart. These protein deposits weaken the heart muscle and, over time, cause severe limitation of heart function, resulting in failure.

Patients with the most prevalent form of ATTR-CM are commonly misdiagnosed in clinics due to a lack of disease awareness and symptoms that mimic other more common heart-related illnesses. Furthermore, individuals face a scarcity of approved medications, as the only treatment particularly suggested for the most frequent form of ATTR-CM costs approximately $225,000 per year and is not widely available.

The ATTRibute-CM phase 3 trial investigators report in the New England Journal of Medicine that the new medicine acoramidis showed potential against ATTR-CM while remaining safe. Their findings indicate that acoramidis achieves near-complete stability of the TTR proteins, potentially slowing or halting disease progression. BridgeBio Pharma (Palo Alto, CA) is pursuing FDA approval for this treatment based on the efficacy and safety findings from this randomized, multicenter, controlled 30-month research.

We hope that, when this drug receives approval from the FDA, it will create competition in the market and drive down the cost of these incredibly expensive medications.”

Daniel Judge, M.D., cardiologist at the Medical University of South Carolina,

ATTRibute-CM investigator and co-leader of the trial’s steering committee

Judge and his colleague Isabella Graef, M.D., of Stanford University, have spent years studying and developing a new medication for these individuals. Graef initiated the development of the novel medicine, which targets misfolded proteins crucial to the condition, and Judge has since been significantly involved in phase 2 and 3 clinical studies, testing the new treatment in his clinic at MUSC. MUSC was one of the first locations in the world to accept patients for the ATTRibute-CM phase 3 trial.

The ATTRibute-CM researchers discovered that acoramidis binds to circulating TTR protein, preventing it from accumulating as amyloid. Compared to a placebo, acoramidis reduced hospitalizations for heart-related events, lowered cardiac congestion as measured by blood tests, and increased the distance walked in six minutes.

According to data presented by Judge at the American Heart Association, five persons required to be treated with acoramidis to prevent one cardiovascular hospitalization in a year during the research. Overall, treatment adverse events were low and comparable between the acoramidis and placebo groups, showing that the medicine is safe. These findings show significant promise for ATTR-CM patients, indicating that acoramidis may slow disease progression and boost survival rates.

Judge, who has over 20 years of experience practicing cardiology and specializes in translational research on heart disease, recruited and tracked numerous patients in the acoramidis clinical trials. He and his co-investigators have seen the innovative medicine progress through translational testing and see the immediate benefit to real patients suffering from ATTR-CM.

“Early in my career, I was disappointed by the lack of any available treatments. I’m glad to see another successful trial for ATTR-CM,” said Judge. “A patient whom I first met in 2017 had a prognosis of three years. Fast forward to 6.5 years later, and this same patient, who was one of the first participants enrolled at MUSC in the phase 2 trial, seems to be getting better due to acoramidis.”

If the illness is stable, there is optimism that the heart will progressively improve. Acoramidis could safely and effectively halt disease progression, improving the quantity and quality of life for ATTR-CM patients globally.