FDA approves acoramidis, BridgeBio’s new ATTR-CM drug
The U.S. Food and Drug Administration (FDA) has approved acoramidis to reduce the risk of cardiovascular death and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
Acoramidis is a selective transthyretin (TTR) stabilizer sold by BridgeBio under the brand name Attruby. It is now the second ATTR-CM medication to gain FDA approval after Pfizer’s tafamidis, which is sold under the names Vyndaqel and Vyndamax.
The FDA’s decision to approve acoramidis is largely based on ATTribute-CM, a clinical trial first published in The New England Journal of Medicine in January.[1] ATTribute-CM randomized more than 600 patients 2:1 to receive acoramidis or a placebo for 30 months. While the overall adverse event rates were comparable for the two treatment arms, acoramidis was still associated with a significant reduction in the risk of serious adverse events.
Researchers presented additional follow-up data from ATTribute-CM at the American Heart Association’s Scientific Sessions 2024 conference in Chicago. Cardiologist Daniel Judge, MD, the chair of cardiovascular research and a professor of medicine at Medical University of South Carolina, presented the new data. In an exclusive video interview, he told Cardiovascular Business that acoramidis was linked to multiple long-term benefits compared to a placebo. In addition, he added, it appears to be more effective the earlier you begin treatment.
BridgeBio, patient advocates celebrate FDA approval
“I’m grateful to each trial participant, their families, and the physicians, scientists and our team at BridgeBio who made this possible,” Neil Kumar, PhD, founder and CEO of BridgeBio, said in a statement. “Our journey is not over as we look to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment.”
“The need for more treatment options for patients living with ATTR-CM is crucial to achieving the goal of better outcomes and improved quality of life,” added Muriel Finkel, president of Amyloidosis Support Groups, a nonprofit organization focused on reaching and helping patients with cardiac amyloidosis. “Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis.”
The good news for BridgeBio does not end with this announcement—the company also received $500 million in royalty payments as a result of this approval.
BridgeBio is now seeking regulatory approval for acoramidis/Attruby in Europe. The company has granted exclusive commercialization rights for the drug to Bayer.
ATTR-CM market getting more competitive
Pfizer’s tafamidis has been the only game in town for treating ATTR-CM since 2019, but BridgeBio’s approval brings new competition to that market. Also, Alynlam Pharmaceuticals recently submitted a request to the FDA to get vutrisiran, which is already approved for the treatment of polyneuropathy of hereditary ATTR amyloidosis, cleared as an ATTR-CM treatment.
“We look forward to working with the FDA over the coming months on this application to bring this medicine to patients as rapidly as possible,” Alnylam Chief Medical Officer Pushkal Garg, MD, said at the time. “Additional filings in other geographies are underway.”
For more context about this approval, watch the full Cardiovascular Business interview with Judge here.
Michael has more than 18 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.