Acoramidis approved to reduce CV death, hospitalization for cardiac amyloidosis
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Key takeaways:
- Acoramidis received FDA approval for reduction of CV death and hospitalization in patients with amyloid cardiomyopathy.
- It is the only agent with a label indicating near-total stabilization of transthyretin.
BridgeBio Pharma announced that acoramidis, its small molecule stabilizer of transthyretin, has been approved by the FDA for the reduction of CV death and hospitalization in patients with transthyretin amyloid cardiomyopathy.
Acoramidis (Attruby) is the first treatment for transthyretin amyloid cardiomyopathy (ATTR-CM) with a label specifying that it achieves near-complete stabilization of transthyretin, according to a press release issued by the company.
The approval was based on the results of the ATTRibute-CM trial, according to the release. As Healio previously reported, in ATTRibute-CM, published in January in The New England Journal of Medicine, a four-step primary hierarchical analysis including all-cause death, CV hospitalization, change from baseline in N-terminal pro-B-type natriuretic peptide level and change from baseline in 6-minute walk distance favored acoramidis over placebo (win ratio, 1.8; 95% CI, 1.4-2.2; P < .001).
“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. Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis,” Muriel Finkel, president of Amyloidosis Support Groups, a nonprofit organization dedicated to the support of amyloidosis patients and caregivers, said in the release.
Daniel P. Judge, MD, FACC, FHFSA, professor of medicine/cardiology, Edwin W. and Teresa H. Rogers Endowed Professor, director of the cardiology fellowship program and director of cardiovascular genetics at Medical University of South Carolina, told Healio that patients with ATTR-CM are currently treated predominantly with tafamidis (Vyndamax, Pfizer), though those who have both hereditary ATTR-CM and polyneuropathy also receive vutrisiran (Amvuttra, Alnylam) or eplontersen (Wainua, AstraZeneca), and people who are stable on those medications will probably stay on them, but “I am enthusiastic, having been involved in phase 2 and phase 3 studies of acoramidis, about starting it for new patients. One factor will be co-pays or other problems patients have run into with tafamidis; like acoramidis, it is well-tolerated, but the main problem with it is financial toxicity. Finally, there is a choice. We don’t have any direct comparisons for the two medications, but I have been really pleased in my experience with both medications. I favor acoramidis as a more potent stabilizer. The inferential data show it seems better at stabilizing the TTR complex. Hopefully, payers will have the opportunity to negotiate and come up with better prices for patients.”
BridgeBio has set up a patient support services program called ForgingBridges to help patients and their families access acoramidis, according to the release.
Editor’s Note: This article was updated on Dec. 2, 2024 to correct the generic name of tafamidis. The Editors regret the error.
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