The U.S. Food and Drug Administration (FDA) has granted approval to BridgeBio Pharma's Attruby (acoramidis), an oral transthyretin (TTR) stabilizer, for the treatment of adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM). This approval marks a significant milestone in the treatment of ATTR-CM, offering a new therapeutic option aimed at reducing cardiovascular mortality and related hospitalizations.
The approval of Attruby was primarily based on the results of the Phase III ATTRibute-CM trial, a randomized, double-blind, placebo-controlled study that enrolled 632 patients with symptomatic ATTR-CM. Participants were randomized in a 2:1 ratio to receive either Attruby or a placebo over a 30-month period. The study's primary endpoint was a composite outcome that included all-cause mortality, frequency of cardiovascular-related hospitalizations, changes in levels of N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and performance on the six-minute walk test.
The ATTRibute-CM trial met its primary endpoint, demonstrating a statistically significant benefit with a win ratio of 1.8. Further supporting Attruby's efficacy, the trial showed a statistically significant treatment effect on the six-minute walk test and the Kansas City Cardiomyopathy Questionnaire, indicating improvements in functional capacity and quality of life. Notably, Attruby also halved the increase in NT-proBNP levels compared to the placebo group, suggesting a reduction in cardiac stress.
According to BridgeBio Pharma CEO and founder Neil Kumar, "With the landmark approval of Attruby, we gain the ability to serve patients with ATTR-CM. I’m grateful to each trial participant, their families and the physicians, scientists and our team at BridgeBio who made this possible." Kumar also noted the company's plans to pursue approvals globally and to continue exploring the full potential of this treatment.
In recognition of the contributions of trial participants in the US, BridgeBio intends to provide Attruby free for life to these patients as a gesture of gratitude. The company has also submitted a marketing authorization application (MAA) to the European Medicines Agency, with a decision expected by 2025. Bayer has secured exclusive rights to commercialize acoramidis for ATTR-CM in Europe.
ATTR-CM is a progressive and often underdiagnosed condition in which the transthyretin protein misfolds and accumulates in the heart, leading to heart failure. Attruby is the first approved product with a label that specifies near-complete TTR stabilization, addressing the underlying cause of the disease. The outcomes from the Phase III ATTRibute-CM trial were published by the company in January 2024.
