BridgeBio Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Attruby™ (acoramidis) for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. This approval marks a significant advancement in the treatment of ATTR-CM, a progressive disease with limited treatment options.
The FDA's decision was based on the positive results from the ATTRibute-CM Phase 3 study, which demonstrated that Attruby significantly reduced death and cardiovascular-related hospitalization, while also improving the quality of life for patients. The study enrolled 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR, who were randomized 2:1 to receive Attruby or placebo for 30 months.
Clinical Efficacy and Safety
The ATTRibute-CM trial met its primary endpoint, demonstrating a statistically significant treatment effect with a Win Ratio of 1.8 (p<0.0001) on a 4-component composite endpoint of all-cause mortality (ACM), cardiovascular hospitalization (CVH), N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and 6-minute walk distance. Notably, Attruby demonstrated a 42% reduction in the composite of ACM and recurrent CVH events relative to placebo at Month 30, and a 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30.
According to Martha Grogan, M.D., of the Mayo Clinic, "Having a new first line treatment option which provides excellent TTR stabilization and improves outcomes in this disease gives patients more options. Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalization as early as three months after initiation of therapy. With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition."
In terms of safety, the most common adverse reactions reported in patients treated with Attruby were diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) compared to placebo. These adverse reactions were generally mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between the Attruby and placebo groups (9.3% and 8.5%, respectively).
Mechanism of Action
Attruby is designed to mimic a naturally occurring "rescue mutation" of the TTR gene (T119M) that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. By achieving near-complete TTR stabilization (≥90%), Attruby preserves the native function of TTR as a transport protein of thyroxine and vitamin A, thereby demonstrating benefits on cardiovascular outcomes.
Patient Support and Access
BridgeBio is committed to ensuring access to Attruby for patients who need it. The company offers a patient support services program called ForgingBridges™, which provides insurance resources, financial assistance options, and a dedicated support team to assist patients in their treatment journey. Furthermore, BridgeBio will provide Attruby free for life to U.S. clinical trial participants, honoring their contribution to the development of this new therapy.
Future Plans
BridgeBio has submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. The company has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe. BridgeBio also plans to pursue approvals in Japan and Brazil, further expanding the global reach of this important new treatment option.
