Acoramidis Gains Global Momentum: FDA Approval, EU Recommendation, and Promising Clinical Data
核心洞察
Acoramidis (Attruby), developed by Stanford Medicine and BridgeBio, receives FDA approval for transthyretin amyloid cardiomyopathy (搜索) (ATTR-CM (搜索)) treatment, marking a significant milestone.
The European Medicines Agency's CHMP recommends acoramidis for EU marketing authorization based on positive Phase 3 ATTRibute-CM trial results.
Clinical trials demonstrate acoramidis' efficacy in reducing cardiovascular-related hospitalizations and improving survival rates for ATTR-CM (搜索) patients.
Acoramidis, a drug initially developed at Stanford Medicine, is gaining global recognition as a promising treatment for transthyretin amyloid cardiomyopathy (搜索) (ATTR-CM (搜索)). The drug has secured FDA approval in the United States and received a positive recommendation from the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) for marketing authorization in the European Union. These regulatory milestones, coupled with compelling clinical trial data, position acoramidis as a potential game-changer in the management of this rare and life-threatening cardiovascular disease.
FDA Approves Acoramidis (Attruby) for ATTR-CM
The U.S. Food and Drug Administration (FDA) approved acoramidis (Attruby) on November 22, 2024, for the treatment of adult patients with ATTR-CM (搜索) to reduce cardiovascular death and cardiovascular-related hospitalization. ATTR-CM is a rare cardiovascular disease characterized by the buildup of misfolded transthyretin (搜索) protein in the heart, leading to stiffness and impaired heart function. The approval marks a significant advancement in the treatment landscape for ATTR-CM, offering a new hope for patients diagnosed with this condition.
Isabella Graef, MD, former faculty member at the Stanford School of Medicine and current CEO of Shenandoah Therapeutics, emphasized the rarity of this accomplishment, stating, "It is an exceedingly rare accomplishment to develop a drug in academia that was approved by the FDA without any further optimization in industry. The FDA approval of Attruby is a testament to the transformative power of rigorous science and team effort within university laboratories."
Positive CHMP Opinion for Acoramidis in the EU
Adding to the momentum, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization in the European Union (EU) for acoramidis for the treatment of wild-type or variant transthyretin (搜索) amyloidosis in adult patients with cardiomyopathy (ATTR-CM (搜索)). The final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the coming months.
Julie Miller Everett, Chief Business Officer for BridgeBio Cardiovascular, expressed enthusiasm about the CHMP's positive recommendation, stating, "We are encouraged by the CHMP’s positive recommendation of acoramidis and the step forward this represents for the patient community. We are excited to work with Bayer to make acoramidis available to patients in Europe upon approval by the European Commission, which we are looking forward to in early 2025."
Clinical Trial Data Supports Acoramidis' Efficacy
The FDA approval and CHMP recommendation are supported by positive results from the Phase 3 ATTRibute-CM study. The study investigated the efficacy and safety of acoramidis given twice daily compared with placebo, in subjects with ATTR-CM (搜索). The study met its primary clinical endpoints by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients in need.
The ATTRibute-CM trial, involving 632 patients, demonstrated that acoramidis significantly reduced the composite endpoint of all-cause mortality and cardiovascular-related hospitalizations compared to placebo (p=0.018). The drug was also associated with a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events, along with a 50% reduction in the cumulative frequency of cardiovascular-related hospitalization events, relative to placebo at month 30.
Collaboration Between Bayer and BridgeBio
Since March 2024, BridgeBio and Bayer have pursued a collaboration for acoramidis. BridgeBio holds the marketing rights for acoramidis in the U.S., while Bayer holds the marketing rights for the product in Europe. This partnership leverages Bayer’s long legacy of expertise in cardiovascular disease and its established European cardiovascular infrastructure paired with BridgeBio’s leadership in the emerging field of ATTR-CM (搜索). Pending European Commission approval, Bayer plans to launch acoramidis in Europe in the first half of 2025.
Acoramidis: A Beacon of Hope for ATTR-CM Patients
With its recent regulatory successes and promising clinical data, acoramidis represents a significant advancement in the treatment of ATTR-CM (搜索). The drug offers a new hope for patients diagnosed with this rare and debilitating cardiovascular disease, potentially improving their survival rates, quality of life, and overall well-being.