FDA Approves BridgeBio's Attruby for ATTR-CM, Reducing Cardiovascular Death and Hospitalization
核心洞察
The FDA has approved Attruby (acoramidis), an oral TTR (搜索) stabilizer developed by BridgeBio, for treating adults with ATTR-CM (搜索).
Attruby demonstrated a significant reduction in cardiovascular death and cardiovascular-related hospitalizations in the Phase 3 ATTRibute-CM study.
Attruby is the first and only approved product with a label specifying near-complete stabilization of transthyretin (搜索) (TTR (搜索)).
BridgeBio Pharma has received FDA approval for Attruby (acoramidis), a novel, orally-administered transthyretin (搜索) (TTR (搜索)) stabilizer, for the treatment of adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM (搜索)). This approval marks a significant advancement in the treatment of ATTR-CM, addressing a critical need to reduce cardiovascular mortality and morbidity associated with the disease.
The FDA's decision was primarily based on the positive outcomes observed in the Phase 3 ATTRibute-CM clinical trial. The study demonstrated that Attruby significantly reduced the risk of cardiovascular death and cardiovascular-related hospitalizations, while also improving the overall quality of life for patients. These findings highlight the potential of Attruby to alter the course of ATTR-CM (搜索), a progressive and often fatal condition.
Mechanism of Action
Attruby is designed to stabilize the TTR (搜索) protein, preventing its misfolding and subsequent aggregation in the heart tissue, which is the hallmark of ATTR-CM (搜索). According to BridgeBio, Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. The drug mimics a naturally occurring 'rescue mutation' of the TTR gene, targeting the root cause of ATTR-CM by stabilizing the native TTR tetramer. This stabilization preserves the native function of TTR as a transport protein for thyroxine and vitamin A, further contributing to its therapeutic benefits.
Clinical Trial Data
The ATTRibute-CM trial was a randomized, double-blind, placebo-controlled study that enrolled patients with ATTR-CM (搜索). The primary endpoint was a composite of all-cause mortality and cardiovascular hospitalization frequency. Detailed results from the trial, including specific hazard ratios and p-values, are expected to be published in a peer-reviewed journal. However, the topline data presented by BridgeBio clearly support the clinical benefit of Attruby in reducing adverse cardiovascular outcomes.
Future Directions
BridgeBio is actively pursuing global approvals for acoramidis, with a Marketing Authorization Application submitted to the European Medicines Agency (EMA). A decision from the EMA is anticipated in 2025. Furthermore, BridgeBio has granted exclusive rights to Bayer for the commercialization of acoramidis in Europe, potentially ensuring broader access to this therapy for patients in need. "With the landmark approval of Attruby, we gain the ability to serve patients with ATTR-CM (搜索)," said Neil Kumar, Ph.D., BridgeBio founder and CEO.
About ATTR-CM
ATTR-CM (搜索) is a progressive disease in which the TTR (搜索) protein misfolds and aggregates, forming amyloid fibrils that deposit in the heart. This leads to thickening of the heart muscle, impaired cardiac function, and ultimately heart failure. The disease is often underdiagnosed, and treatment options have been limited, underscoring the importance of new therapies like Attruby.