European Commission Approves BEYONTTRA™ (acoramidis) as First ≥90% TTR Stabilizer for ATTR-CM

PALO ALTO, CA, USA I February 11, 2025 I BridgeBio Pharma, Inc., a pioneering biopharmaceutical firm focused on genetic disorders, has announced a significant milestone. The European Commission has granted marketing authorization for acoramidis, branded as BEYONTTRA™, in the European Union (EU). This approval pertains to the treatment of transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM). This condition, characterized by an aggressive progression and adverse prognosis if untreated, now has a new treatment option. Acoramidis functions as a selective small molecule that stabilizes transthyretin (TTR) by more than 90%, offering a promising therapeutic approach for this serious disease.

ATTR-CM is a life-threatening disorder that causes restrictive cardiomyopathy, eventually leading to heart failure. The newly approved treatment, acoramidis, has shown a marked improvement in reducing mortality and decreasing cardiovascular hospitalizations in as little as three months. Bayer has been designated as responsible for all commercial activities concerning acoramidis in the EU.

Dr. Marianna Fontana, a Cardiology Professor at University College London, emphasized the significance of this approval. According to her, providing an effective treatment option for those suffering from ATTR-CM in the EU is a substantial achievement, offering hope to patients with limited alternatives.

The EU's approval was heavily based on the findings from the pivotal ATTRibute-CM Phase 3 trial. This study involved 632 participants diagnosed with symptomatic ATTR-CM stemming from either wild-type or variant TTR. Participants were randomly assigned to receive either acoramidis or a placebo over a 30-month period. The results were promising—acoramidis met its primary endpoints by significantly decreasing cardiovascular-related hospitalizations, improving survival rates, and maintaining patients' functional capacity and quality of life.

Dr. Jonathan Fox, Chief Medical Officer of BridgeBio Cardiorenal, expressed that the EU approval marks a crucial advancement for ATTR-CM patients desperately needing innovative treatments. He acknowledged the invaluable contributions from trial participants, their families, and the medical professionals involved in the clinical program. Dr. Fox looked forward to collaborating with Bayer to serve the needs of ATTR-CM patients across the EU.

Following the EU authorization, Bayer is set to launch acoramidis in the first half of 2025. The drug had previously received approval from the U.S. FDA in November 2024, under the name Attruby™, with a label underscoring its near-complete TTR stabilization. Since then, BridgeBio has experienced robust commercial momentum in the U.S., with numerous prescriptions being issued.

A collaboration between BridgeBio and Bayer was initiated in March 2024, giving Bayer exclusive rights to commercialize acoramidis in the EU. The terms of this agreement include a $75 million milestone payment to BridgeBio following the European Commission's approval, along with tiered royalties on sales.

Currently, acoramidis is undergoing evaluation for approval by regulatory agencies in Japan and Brazil. Meanwhile, BEYONTTRA has been generally well-tolerated in clinical trials, with mild side effects like diarrhea and abdominal pain that resolved without the need to discontinue the medication.

BridgeBio Pharma, Inc., founded in 2015, is committed to developing groundbreaking medicines for genetic diseases. With a pipeline that spans from early research to advanced clinical trials, BridgeBio is dedicated to delivering innovative genetic therapies to provide relief for patients worldwide.