BridgeBio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Core Insights - The FDA has accepted BridgeBio Pharma's New Drug Application (NDA) for acoramidis, with an action date set for November 29, 2024, and no advisory committee meeting planned [1] - The European Medicines Agency (EMA) has also accepted the Marketing Authorization Application for acoramidis, with plans for additional global regulatory submissions [1][3] - In the ATTRibute-CM study, acoramidis showed an 81% absolute survival rate and a mean annual cardiovascular-related hospitalization frequency of 0.29, indicating significant clinical benefits [1][2] Regulatory Developments - The FDA's acceptance of the NDA reinforces the potential of acoramidis to contribute significantly to the treatment of patients with ATTR-CM [2] - The company is preparing for further regulatory submissions in other countries and regions following the EMA's acceptance [3] Clinical Study Results - The ATTRibute-CM study reported a Win Ratio of 1.8 (p<0.0001) on the primary endpoint, indicating highly statistically significant results [2] - Acoramidis was well-tolerated with no safety concerns identified during the clinical trials [2] Company Overview - BridgeBio Pharma is a commercial-stage biopharmaceutical company focused on developing transformative medicines for genetic diseases and cancers [4] - Founded in 2015, the company aims to leverage advances in genetic medicine to expedite patient access to new treatments [4]