In a significant development for cardiovascular medicine, BridgeBio’s Attruby (acoramidis) has received FDA approval for treating Transthyretin amyloid cardiomyopathy (ATTR-CM) in adults, marking a crucial milestone for a rare and severe heart condition that can lead to fatal outcomes if left untreated. ATTR-CM, which involves the deposition of amyloid proteins in the heart, can result in heart failure and death. With the introduction of Attruby, the first approved treatment to provide near-complete stabilization of Transthyretin (TTR), patients have new hope for managing this debilitating disease. This groundbreaking approval is underpinned by the successful ATTRibute-CM Phase III study, which demonstrated that Attruby significantly decreased mortality rates and cardiovascular-related hospitalizations, while also enhancing patients’ quality of life. What stands out is that these considerable benefits were observed as early as three months into the treatment, showcasing the drug’s rapid efficacy.
Groundbreaking Study and Clinical Efficacy
The ATTRibute-CM Phase III study, which played a pivotal role in securing FDA approval for Attruby, involved 632 symptomatic patients randomized to receive either the drug or a placebo over a period of 30 months. The results were compelling; patients treated with Attruby exhibited markedly improved outcomes in multiple health metrics. For instance, the study reported a significant treatment effect on both the Kansas City Cardiomyopathy Questionnaire and a six-minute walk test, along with a substantially lower increase in NT-proBNP levels compared to the placebo group. These metrics are critical indicators of heart function and overall well-being, and the improvements underscore Attruby’s effectiveness in addressing ATTR-CM.
Experts in the field have highlighted the importance of this development. Dr. Martha Grogan from the Mayo Clinic emphasized the significance of having a new first-line treatment option. She noted that Attruby not only provides excellent TTR stabilization but also markedly improves patient outcomes. The drug works by mimicking a naturally occurring stabilizing mutation in the TTR gene, preserving its vital function as a transport protein for thyroxine and vitamin A. The positive findings of the study were published in The New England Journal of Medicine, cementing the credibility of the results and providing a robust basis for the FDA’s approval decision.
Implications for the Future and Global Approvals
The approval of Attruby marks a major breakthrough in treating ATTR-CM, offering new patient care options previously unavailable. BridgeBio isn’t limiting this advancement to the U.S.; they’re also pursuing approval from the European Medicines Agency, expecting a decision in 2025. This effort highlights their goal of making this innovative treatment accessible globally. BridgeBio’s ForgingBridges program further exemplifies their commitment by helping U.S. patients access Attruby, ensuring financial barriers don’t obstruct treatment.
Muriel Finkel, president of Amyloidosis Support Groups, emphasized the profound impact of more treatment options. She believes Attruby brings hope and significantly improves the quality of life for amyloidosis patients. This positive change in treatment approaches is expected to inspire more research and development, leading to further advancements. BridgeBio’s CEO, Neil Kumar, expressed gratitude to trial participants and reiterated the company’s dedication to obtaining approvals in Europe, Japan, and Brazil.
In summary, the FDA’s approval of Attruby represents a significant leap forward in combating Transthyretin amyloid cardiomyopathy. It not only demonstrates the drug’s ability to improve clinical outcomes but also sets the stage for global accessibility and ongoing innovation in the field. The journey from clinical trials to approval has created new hope and enhanced quality of life for patients worldwide, marking a milestone for BridgeBio and the entire medical community in addressing this challenging condition.
