BridgeBio Pharma, Inc. (Nasdaq: BBIO) has achieved a significant milestone by filing a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for acoramidis, a potential treatment for ATTR-CM (transthyretin amyloid cardiomyopathy). The submission is based on positive results from the Phase 3 study, ATTRibute-CM, which assessed the efficacy and safety of acoramidis—an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR).

Jonathan Fox, President and Chief Medical Officer of BridgeBio Cardiorenal, underscored the importance of this NDA submission, emphasizing the consistent positive evidence supporting acoramidis's potential to offer clinical benefits surpassing current therapeutic options for ATTR-CM patients. Fox expressed gratitude to all participants, their families, investigators, collaborators, and the BridgeBio team for their contributions to this achievement.

In July 2023, BridgeBio reported highly statistically significant results from the ATTRibute-CM study, demonstrating a Win Ratio of 1.8 (p<0.0001) on the primary endpoint. Acoramidis exhibited good tolerability, with no identified safety signals of clinical concern. Additional analyses presented at the European Society of Cardiology Congress 2023 and the American Heart Association Scientific Sessions 2023 showcased positive outcomes, including an 81% absolute survival rate on treatment.

The FDA will conduct a 60-day filing review to determine the completeness and acceptance of the NDA for review. BridgeBio plans to submit additional marketing authorization applications to global health authorities in 2024, furthering its commitment to making acoramidis available as a treatment option for ATTR-CM patients worldwide.

 

Source: globenewswire.com

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