FDA Approves BridgeBio’s Attruby for Treating ATTR-CM

The US Food and Drug Administration (FDA) has given the green light to BridgeBio Pharma’s newest drug, Attruby (acoramidis), aimed at treating adults with transthyretin amyloid cardiomyopathy (ATTR-CM). This oral transthyretin (TTR) stabilizer is expected to reduce cardiovascular-related deaths and hospitalizations among patients.

Attruby stands out as the first product to receive FDA approval with a label indicating near-complete TTR stabilization. As a result of this approval, BridgeBio Pharma will receive a $500 million payment under a royalty funding agreement.

The FDA’s decision relied heavily on the results of the ATTRibute-CM Phase III trial, which included 632 participants suffering from symptomatic ATTR-CM. They were randomly divided in a 2:1 ratio to either receive Attruby or a placebo over a 30-month period. The primary endpoint of the trial was a composite of all-cause mortality, cardiovascular-related hospitalizations, levels of N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and the six-minute walk test. The trial achieved its primary endpoint with a win ratio of 1.8.

Attruby exhibited a significant positive effect, particularly noted in the six-minute walk test, as well as in the Kansas City Cardiomyopathy Questionnaire. Additionally, the treatment notably reduced the increase in NT-proBNP levels by half compared to the placebo group.

In appreciation of the participants’ contributions, BridgeBio Pharma has announced that those in the US who took part in the trial will receive Attruby for free for life. This gesture acknowledges their role in the drug’s development and approval.

Neil Kumar, CEO and founder of BridgeBio Pharma, expressed gratitude to everyone involved in the trial, including patients, their families, and the medical and scientific teams. He emphasized that the approval of Attruby allows the company to serve patients affected by ATTR-CM and highlighted the ongoing efforts to seek approvals in other regions such as Europe, Japan, and Brazil. BridgeBio Pharma is also aiming to further explore the treatment’s full potential and is committed to continuing its patient-first mission. This marks the company's third FDA approval in less than a decade.

BridgeBio Pharma has also submitted a marketing authorization application to the European Medicines Agency, with an expected decision by 2025. Furthermore, Bayer has obtained exclusive rights to commercialize acoramidis for treating ATTR-CM in Europe.

The results of the Phase III ATTRibute-CM trial were published by BridgeBio Pharma in January 2024, detailing the significant findings and the positive impact of Attruby on patients with ATTR-CM. The approval of Attruby represents a significant milestone for BridgeBio Pharma and offers new hope for patients with this challenging condition.