BridgeBio’s BEYONTTRA (Acoramidis) Approved In The UK for ATTR-CM, Marking A Major Advance In Cardiomyopathy Care.

BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for BEYONTTRA® (acoramidis), a near-complete (≥90%) transthyretin (TTR) stabilizer, for the treatment of wild-type or variant transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adult patients.

This approval makes BEYONTTRA the first and only ATTR-CM treatment approved in the U.S., EU, UK, and Japan with a label specifying near-complete TTR stabilization. Acoramidis is a selective, orally administered small molecule designed to stabilize TTR, helping prevent the progression of ATTR-CM, a progressive, fatal disease that leads to restrictive cardiomyopathy and heart failure. Bayer will oversee the commercialization of BEYONTTRA in the UK.

The MHRA’s decision is based on results from the pivotal Phase 3 ATTRibute-CM study, where acoramidis demonstrated the most rapid benefit seen in any Phase 3 trial for ATTR-CM to date. In the study of 632 participants, those treated with acoramidis showed a separation in the time to first event — defined as all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH) — as early as three months compared to placebo. At month 30, acoramidis reduced the composite of ACM and recurrent CVH events by 42% and reduced the cumulative frequency of CVH events by 50% compared to placebo.

Dr. Julian Gillmore, M.D., Ph.D., from University College London's Centre for Amyloidosis, emphasized the significance of this approval, stating, “ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients’ quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied, and associated with low awareness, frequently resulting in delayed or completely missed diagnosis, which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalizations and escalating healthcare costs and is ultimately fatal."
He added, “The UK authorization of BEYONTTRA is welcome news for eligible patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.”

The ATTRibute-CM study successfully met its primary clinical endpoints by improving survival, reducing cardiovascular hospitalizations, and preserving functional capacity and quality of life. These findings underline the potential of BEYONTTRA to offer meaningful disease modification for ATTR-CM patients.

Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal, highlighted the importance of the milestone, stating, “We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to BEYONTTRA since they are in great need of new disease-modifying treatments for their condition.” He further expressed gratitude, saying, “We appreciate the time and commitment of every clinical trial participant and their families, and the dedicated support of the physicians and scientists involved in the clinical program. This important milestone would not have been possible without their commitment to the program.” Dr. Fox also confirmed that BridgeBio looks forward to expanding its collaboration with Bayer to serve ATTR-CM patients across the UK and Europe, while working towards broader global access.