BridgeBio Pharma announced that the U.S. FDA has approved its orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.
Acoramidis tablets, branded Attruby, are the first and only approved products with a label specifying near-complete stabilization of TTR. When TTR proteins are destabilized, they can build up to form amyloid deposits. These deposits cause thickening and stiffening of the heart walls, impairing the heart's ability to pump blood efficiently, which can result in heart failure. Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.
Approval was based on the on positive results seen in the ATTRibute-CM phase 3 study, where Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life. The study, which enrolled 632 participants with symptomatic ATTR-CM, Attruby demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and six-minute walk test. Additionally, the increase in NT-proBNP on treatment was about half that of placebo.
BridgeBio says it will provide all U.S. clinical trial patients Attruby free for life. The company will now pursue approvals globally, starting with Europe, Japan and Brazil.
Pfizer won the first approval in the lucrative ATTR-CM cardiomyopathy space in 2019, when the FDA approved Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by ATTR-CM in adults. Tafamidis sales hit $3.3 billion last year. Massachusetts-based Alnylam also has an ATTR-CM cardiomyopathy drug, vutrisiran, currently in phase 3 trials.