Acoramidis

PALO ALTO, CA, USA I February 11, 2025 I BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the European Commission has granted marketing authorization in the European Union (EU) for acoramidis, under the brand name BEYONTTRA™, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered near-complete (≥90%) transthyretin (TTR) stabilizer. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Bayer will be responsible for all commercial activity for acoramidis in the EU. “ATTR-CM is a rapidly progressing disease with a poor prognosis when left untreated, making the approval of acoramidis, which has demonstrated improved benefit on all-cause mortality and cardiovascular-related hospitalizations in as few as three months, a very important accomplishment for patients. We are pleased that people living with ATTR-CM will have access to another treatment option in the EU,” said Marianna Fontana, M.D., Ph.D., Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London. The approval in the EU is based on results of the pivotal ATTRibute-CM Phase 3 study of acoramidis, which showed clear benefits on cardiovascular outcomes. ATTRibute-CM evaluated the efficacy and safety of acoramidis in 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR who were randomized 2:1 to receive acoramidis or placebo for 30 months. The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients in need. “The EU approval of acoramidis is a significant advancement for patients living with ATTR-CM in need of new disease-modifying treatments for their condition,” said Dr Jonathan Fox, BridgeBio Cardiorenal Chief Medical Officer. “This approval would not have been possible without the commitment of the clinical trial participants and their families, and the dedicated support of the physicians and scientists involved in the clinical program. Alongside our able partners at Bayer we look forward to this new opportunity to serve ATTR-CM patients across the European Union.” Following EU approval, Bayer will launch acoramidis in the first half of 2025. Acoramidis was approved as Attruby™ by the U.S. FDA in November 2024 with a label specifying near-complete stabilization of TTR. As reported on January 13, BridgeBio has seen strong commercial momentum, with 430 patient prescriptions written by 248 physicians since the U.S. approval. In March 2024, BridgeBio and Bayer initiated a collaboration for acoramidis, which granted Bayer exclusive commercialization rights in the EU. Based on terms of the licensing agreement, BridgeBio will receive a $75 million milestone payment upon European Commission approval. BridgeBio will also receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in the EU following initiation of commercialization efforts. Acoramidis is currently under review for approval by the Japanese Pharmaceuticals and Medical Devices Agency and the Brazilian Health Regulatory Agency . 1 Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. About BEYONTTRA BEYONTTRA is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). BEYONTTRA was generally well-tolerated. The most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation. For full prescribing information, please refer to the Summary of Product Characteristics (SmPC) which will be available on the European Medicines Agency (EMA) website once published. About BridgeBio Pharma, Inc. BridgeBio Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn , Twitter and Facebook . SOURCE: BridgeBio Pharma

The therapy has demonstrated significant benefits in cardiovascular outcomes. Credit: Jo Panuwat D/Shutterstock. The European Commission (EC) has granted marketing authorisation to BridgeBio Pharma’s selective small molecule, acoramidis, under the brand name Beyonttra, for treating wild-type or variant transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM). An orally administered near-complete (≥90%) transthyretin (TTR) stabiliser, acoramidis is designed to combat this progressive fatal disease, which can lead to heart failure. The approval in the European Union (EU) is based on outcomes from the Phase III ATTRibute-CM trial of the therapy, which demonstrated cardiovascular benefits. The study involved 632 subjects with symptomatic ATTR-CM and met its primary clinical endpoints at month 30. It showed a significant decrease in cardiovascular-related hospitalisation, improved survival rates, preserved functional capacity and better quality of life for individuals. BridgeBio Cardiorenal chief medical officer Dr Jonathan Fox stated: “The EU approval of acoramidis is a significant advancement for patients living with ATTR-CM in need of new disease-modifying treatments for their condition. “This approval would not have been possible without the commitment of the clinical trial participants and their families, and the dedicated support of the physicians and scientists involved in the clinical programme.” Bayer, responsible for all commercial activities for the therapy in the EU, is set to launch Beyonttra in the first half of 2025. BridgeBio and Bayer initiated a partnership for the therapy in March 2024, granting the latter exclusive commercialisation rights in the EU. Acoramidis is also under review for approval by the Brazilian Health Regulatory Agency and the Japanese Pharmaceuticals and Medical Devices Agency. The latest development follows the therapy’s approval under the name of Attruby by the US Food and Drug Administration in November 2024 to treat adults with ATTR-CM.