FDA Approves BridgeBio Pharma's ATTR-CM Treatment

By Tamara Parker • Monday, November 25 16:34 • Permanent Link

BridgeBio Pharma, Inc. (NASDAQ: BBIO) recently announced that the U.S. Food and Drug Administration (FDA) has approved attruby™ (acoramidis), a near-complete stabilizer of transthyretin (TTR), for the treatment of adults with transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. This approval is based on positive results from the attribute-cm phase 3 study, which enrolled 632 participants with symptomatic ATTR-CM.

The results from the study demonstrated significant improvements with attruby compared to placebo, including a 42% reduction in composite all-cause mortality (ACM) and recurrent cardiovascular-related hospitalization (CVH) events relative to placebo at month 30. Additionally, there was a 50% reduction in the cumulative frequency of CVH events relative to placebo at month 30. Notably, the time to first event (ACM or CVH) durably separated relative to placebo in as few as 3 months.

In terms of safety, adverse reactions such as diarrhea and upper abdominal pain were reported in patients treated with attruby, with the majority of these reactions being mild and resolving without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with attruby and placebo.

BridgeBio will receive a $500 million payment under their royalty funding agreement with this approval. The company also plans to share management remarks on key aspects of the attruby label and important access programs on Friday, November 22, 2024, at 8:00 pm ET.

Neil Kumar, Ph.D., the founder and CEO of BridgeBio, expressed gratitude to the trial participants, their families, physicians, scientists, and the company's team for making this milestone possible. He also highlighted the company's commitment to pursuing global approvals and continuing to explore the full potential of this treatment.

BridgeBio has also submitted a marketing authorization application to the European Medicines Agency, with a decision expected in 2025, and has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.

Following these announcements, the company's shares moved 16.1%, and are now trading at a price of $27.19. Check out the company's full 8-K submission here.

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