FDA Approves BridgeBio's Acoramidis (ATTRUBY) for Transthyretin Amyloid Cardiomyopathy
Key Insights
The FDA has approved acoramidis (ATTRUBY), an oral transthyretin (search) stabilizer, for treating transthyretin amyloid cardiomyopathy (search) (ATTR-CM (search)) in adults, aiming to reduce cardiovascular death and hospitalization.
Approval was based on the Phase 3 ATTRibute-CM study, which demonstrated significant reductions in mortality and cardiovascular hospitalizations, along with improved patient quality of life.
BridgeBio will receive a $500 million payment due to the approval, and a decision from the European Medicines Agency on their marketing application is expected in 2025.
BridgeBio Pharma Inc. has received FDA approval for ATTRUBY (acoramidis), an orally administered near-complete stabilizer of transthyretin (search) (TTR), for the treatment of adult patients with transthyretin amyloid cardiomyopathy (search) (ATTR-CM (search)). This approval marks a significant advancement in the treatment of ATTR-CM, a progressive and often fatal disease characterized by the accumulation of misfolded TTR protein in the heart. The drug is indicated to reduce cardiovascular mortality and cardiovascular-related hospitalizations in this patient population.
The FDA's decision was primarily based on the positive results from the ATTRibute-CM Phase 3 study. The study demonstrated that acoramidis significantly reduced all-cause mortality and cardiovascular-related hospitalizations compared to placebo. Furthermore, patients treated with acoramidis experienced improvements in quality of life, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ).
ATTR-CM (search) is a systemic disease with substantial unmet need. The accumulation of misfolded TTR protein leads to restrictive cardiomyopathy, heart failure, and ultimately, death. Current treatment options are limited, and acoramidis represents a novel approach by stabilizing the TTR protein and preventing its misfolding and aggregation.
Under a royalty funding agreement, BridgeBio is set to receive a $500 million payment following the FDA approval. The company has also submitted a Marketing Authorization Application to the European Medicines Agency, with a decision anticipated in 2025. BridgeBio has granted exclusive rights to Bayer for the commercialization of acoramidis in Europe.
The ATTRibute-CM trial was a randomized, double-blind, placebo-controlled study that enrolled 632 patients with ATTR-CM (search). Patients were randomized to receive either acoramidis 800 mg twice daily or placebo, in addition to standard of care. The primary endpoint was a hierarchical analysis of all-cause mortality, frequency of cardiovascular hospitalization, change from baseline in 6-minute walk test distance, and change from baseline in KCCQ overall summary score. Key secondary endpoints included all-cause mortality and cardiovascular hospitalization analyzed separately.
Acoramidis offers a convenient oral administration route, distinguishing it from other available treatments that require intravenous infusion. This may improve patient adherence and convenience. The drug's mechanism of action, stabilizing TTR, directly addresses the underlying cause of ATTR-CM (search), offering a potential disease-modifying effect.