The European Commission has granted marketing authorization in the European Union (EU) for BridgeBio Pharma’s 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.
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.
