UK MHRA Approves BridgeBio's Acoramidis for ATTR-CM

BridgeBio Pharma has received marketing authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its drug acoramidis, marketed as Beyonttra. This medication is designed to treat adult patients suffering from wild-type or variant transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a serious condition that often results in restrictive cardiomyopathy and heart failure.

Acoramidis is administered orally and functions as a selective small molecule that stabilizes transthyretin (TTR) protein by over 90%. This stabilization helps mitigate the progression of the disease, offering significant cardiovascular benefits to patients. The approval from the MHRA was largely based on the positive results from the ATTRibute-CM Phase III clinical trial. This pivotal study involved 632 participants with symptomatic ATTR-CM, who received either the drug or a placebo over a 30-month period.

The trial successfully met its primary clinical endpoints by the end of the 30 months, showcasing a reduction in hospitalizations related to cardiovascular issues. Additionally, it preserved the functional capacity of the patients, improved their survival rates, and enhanced their overall quality of life. These outcomes underscore the efficacy and safety of acoramidis in managing this debilitating condition.

Previously, the US Food and Drug Administration (FDA) had approved the drug under the name Attruby in November 2024, while the European Commission (EC) followed suit, granting approval in February 2025 under the name Beyonttra. Additionally, the Ministry of Health, Labour, and Welfare in Japan approved the therapy in March 2025.

In the UK, the commercial activities associated with Beyonttra will be managed by Bayer. This follows a partnership agreement established in March 2024, where BridgeBio Pharma granted Bayer exclusive commercial rights for the therapy in European markets. The licensing agreement includes BridgeBio receiving tiered royalties starting in the low-thirties percent from sales in the UK once commercialization begins.

Jonathan Fox, the chief medical officer and president of BridgeBio Cardiorenal, expressed pride in achieving another regulatory approval for acoramidis. He highlighted the importance of providing UK patients with access to Beyonttra, emphasizing the urgent need for new treatments that can effectively alter the disease course of ATTR-CM. Fox also mentioned the company's intention to expand its collaboration with Bayer to support ATTR-CM patients not only in the UK but across Europe and other regions worldwide.

With this approval, BridgeBio Pharma aims to bring a much-needed therapeutic option to patients suffering from this life-threatening condition, offering hope for improved management of their disease. The successful partnership with Bayer promises to facilitate the distribution and availability of Beyonttra across multiple territories, thereby addressing the significant unmet medical needs of ATTR-CM patients globally.