Beyonttra™ (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved in Japan to Treat ATTR-CM

Core Viewpoint - BridgeBio Pharma has received approval from the Japanese Ministry of Health for acoramidis, branded as Beyonttra, to treat adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), marking a significant advancement in treatment options for this progressive and fatal disease [1][2]. Company Overview - BridgeBio Pharma, Inc. is a biopharmaceutical company focused on developing treatments for genetic diseases, founded in 2015. The company aims to deliver transformative medicines through a pipeline that includes early science to advanced clinical trials [9]. Product Details - Acoramidis is a selective small molecule, orally administered, and is a near-complete (≥90%) stabilizer of transthyretin (TTR). It has shown potential in reducing all-cause and cardiovascular mortality risks [1][6]. - The drug was well-tolerated in clinical trials, with a reported 0% mortality during a 30-month treatment period in the Japanese Phase 3 study [3][8]. Clinical Trial Results - The approval in Japan was based on positive outcomes from a Phase 3 open-label study and the global ATTRibute-CM Phase 3 trial. Key findings include: - Early separation in time to first event (all-cause mortality or cardiovascular-related hospitalization) at 3 months compared to placebo [3][8]. - A 42% reduction in composite all-cause mortality and recurrent cardiovascular hospitalization events at Month 30 relative to placebo [3][8]. - A 50% reduction in cumulative frequency of cardiovascular hospitalization events at Month 30 compared to placebo [3][8]. Commercialization and Financials - Alexion, AstraZeneca Rare Disease holds an exclusive license to develop and commercialize acoramidis in Japan. BridgeBio will receive a $30 million milestone payment upon approval and low double-digit royalties on sales [4][8].