Novel heart failure drug could halt disease progression

The small molecule heart failure drug acoramidis has potential in slowing or stopping the progression of transthyretin amyloid cardiomyopathy (ATTR-CM), according to study data published in the New England Journal of Medicine.

Findings from the Phase III trial ATTRibute-CM showed the novel drug is safe and can achieve near-complete stabilisation of TTR proteins, which play a key role in disease progression of ATTR-CM. This is because acoramidis stops these proteins from depositing as amyloid protein.

Study findings

Compared with placebo, acoramidis was found to reduce the number of patients who were hospitalised due to a heart-related event. The drug was also shown to lower cardiac congestion. Additionally, patients increased their walking distance within a specified period of six minutes, according to the researchers.

There were five people who needed acoramidis treatment to prevent one cardiovascular hospitalisation in one year, data presented at the American Heart Association (AHA) revealed.

Overall, with a low rate of treatment adverse events and with similar occurrence in individuals given acoramidis and placebo, this suggests that the drug is safe, the researchers highlighted.

As such, by facilitating disease stabilisation, the researchers stated there is hope that the heart can improve gradually. As a result, halting disease progression safely and effectively using acoramidis could increase the quantity and quality of life for those with transthyretin amyloid cardiomyopathy.

Regulatory developments for heart failure drug acoramidis

Last month, BridgeBio Pharma confirmed that it had that submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for acoramidis to treat transthyretin amyloid cardiomyopathy, based on data from the Phase III trial.

“We hope that, when this drug receives approval from the US FDA, it will create competition in the market and drive down the cost of these incredibly expensive medications,” noted Dr Daniel Judge, a cardiologist at the Medical University of South Carolina, an ATTRibute-CM investigator and co-leader of the trial’s steering committee.