The US health authority FDA has given the company BridgeBio Pharma the green light for its drug Attruby (Acoramidis), which is now approved for the treatment of adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This medication is characterized by its ability to achieve nearly complete stability of the TTR protein and aims to reduce cardiovascular deaths and associated hospitalizations.

The approval of Attruby triggers a payment of 500 million dollars to BridgeBio under a licensing agreement. The approval was based on the ATTRibute-CM phase III study, which examined 632 participants with symptomatic ATTR-CM. In this study, participants were treated in a 2:1 ratio with either Attruby or a placebo over 30 months. The results were impressive: the primary goals of the study—including overall mortality, cardiovascular-related hospitalizations, and biochemical markers—were achieved with a win ratio of 1.8.

Particularly noteworthy was the significant improvement in the six-minute walk test and the Kansas City Cardiomyopathy Questionnaire, which set Attruby apart from its competitors. Additionally, the increase in NT-proBNP levels was halved compared to the placebo group, underscoring the drug's efficacy. As a special thank you to the study participants in the US, BridgeBio Pharma plans to offer them a lifelong free supply of Attruby.

Neil Kumar, CEO and founder of BridgeBio, expressed his gratitude and vision for the future: "The groundbreaking approval of Attruby allows us to better care for patients with ATTR-CM. This is our third FDA product within less than ten years. Our journey continues with the global approval of Attruby—primarily in Europe, Japan, and Brazil."

A corresponding market authorization application has been submitted to the European Medicines Agency, with a decision expected by 2025. In addition, Bayer has acquired exclusive rights to market Acoramidis for the treatment of ATTR-CM in Europe. The impressive results of the ATTRibute-CM study were already published in January 2024.