FDA Approves BridgeBio’s Attruby: A Breakthrough Treatment for ATTR-CM

FDA Approves BridgeBio’s Attruby: A Breakthrough Treatment for ATTR-CM

In a significant medical breakthrough, the Food and Drug Administration (FDA) has approved BridgeBio Pharma, Inc.’s Attruby (acoramidis) for the treatment of adults with transthyretin amyloid cardiomyopathy (ATTR-CM). This orally administered drug represents a major step forward in managing this rare and often fatal cardiovascular disease. Attruby achieves near-complete (≥90%) stabilization of transthyretin (TTR), a protein whose misfolding is the root cause of ATTR-CM.

The FDA’s approval is firmly grounded in the robust results of the ATTRibute-CM Phase 3 clinical trial. This study, involving 632 participants with symptomatic ATTR-CM, demonstrated that Attruby significantly reduced both cardiovascular-related deaths and hospitalizations. Furthermore, the trial indicated a marked improvement in patients’ quality of life, offering tangible hope for those battling this debilitating condition.

Attruby stands out as the first and only approved therapy specifically labeled for near-complete TTR stabilization. Its innovative design mimics a naturally occurring, protective mutation of the TTR gene (T119M), directly addressing the underlying cause of ATTR-CM—the destabilization of the TTR tetramer. This near-complete stabilization not only prevents the harmful effects of misfolded TTR but also maintains the protein’s vital function as a transporter of thyroxine and vitamin A, contributing to its positive impact on cardiovascular outcomes.

The approval has generated considerable excitement within the pharmaceutical industry. BridgeBio has already submitted a Marketing Authorization Application to the European Medicines Agency (EMA), anticipating a decision in 2025. Furthermore, a licensing agreement with Bayer AG grants Bayer exclusive rights to commercialize acoramidis for ATTR-CM within Europe.

Market Dynamics and Competitive Landscape

The approval of Attruby immediately places it in competition with Pfizer’s tafamidis, another established treatment for ATTR-CM. William Blair analysts highlight the significance of all-cause mortality reduction in achieving substantial market share. While Attruby’s label currently doesn’t include data on overall mortality, Pfizer may leverage its mortality benefit claims in marketing efforts. This competitive dynamic underscores the importance of ongoing research and the need for continued monitoring of both treatments’ long-term effects.

BridgeBio’s commitment to patient care is evident in its decision to provide free Attruby to all participants who contributed to the success of the ATTRibute-CM trial. This demonstrates a dedication to both advancing medical science and ensuring equitable access to life-changing therapies.

Stock Market Reaction

The FDA’s approval has sent positive ripples through the financial markets. BBIO stock experienced a remarkable 24.3% surge in premarket trading on Monday, reaching $29.10 per share. This signifies investor confidence in Attruby’s potential to revolutionize ATTR-CM treatment and represents a significant milestone for BridgeBio Pharma.

The FDA’s approval of Attruby marks not only a victory for BridgeBio but a beacon of hope for individuals battling ATTR-CM. This innovative therapy promises a brighter future for patients and represents a substantial step forward in the treatment of this rare and challenging disease.

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