FDA Accepts Filing of BridgeBio Pharma’s New Drug Application for an ATTR-CM Treatment

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The biopharma company’s Phase 3 trial for its acoramidis product generated statistically significant results.

Cardiac muscle fibers in the myocardium. Image Credit: Adobe Stock Images/JosLuis.com

Cardiac muscle fibers in the myocardium. Image Credit: Adobe Stock Images/JosLuis.com

The FDA has accepted the filing of BridgeBio Pharma, Inc.’s new drug application (NDA) for acoramidis, an investigational drug used for the treatment of ATTR-CM.1 Also known as transthyretin amyloid cardiomyopathy, the disease is caused by misfolded transthyretin (TTR) protein getting deposited into the myocardium.2

This decision was made after positive results of ATTRibute-CM, the commercial-stage biopharmaceutical company’s Phase 3 study that set out to determine the efficacy and safety of acoramidis, an investigational, orally-administered, highly potent, small molecule stabilizer of TTR. Under the Prescription Drug User Fee Act (PDUFA), the FDA scheduled an action date of Nov. 29, 2024, but is not planning to hold an advisory committee meeting to examine the application.

BridgeBio first announced the positive results from ATTRibute-CM back in July 2023,3 indicating that it was a highly statistically significant result, due to a win ratio of 1.8 (p<0.0001) on the primary endpoint (which is a hierarchical analysis that arranged in order: ACM, followed by frequency of cardiovascular-related hospitalization (CVH), then change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP), then change from baseline in a six-minute walk distance (6MWD)). Acoramidis was described as “well-tolerated,” displaying no signs of any possible clinical concern. It also demonstrated an absolute survival rate of 81%, along with a 0.29 observed mean annual CVH frequency.

“The FDA’s acceptance of our NDA submission for review reinforces our belief in acoramidis and its potential to make an important contribution to the care of patients with ATTR-CM,” says Jonathan Fox, MD, PhD, president and chief medical officer of BridgeBio Cardiorenal. “We look forward to the upcoming review process and the potential for approval in the United States. Similarly, with the European marketing authorization application accepted and with plans to extend our submissions to other countries and regions, we are committed to making acoramidis available to patients.”

Isabelle Lousada, president and CEO of the Amyloidosis Research Consortium, a global nonprofit organization that strives to further developments in amyloidosis, feels that this NDA filing could be a step in the right direction in the ATTR-CM battle.

“As part of our mission, we seek to improve the lives of patients with amyloidosis by providing support to them and their caregivers throughout their journey,” she notes. “There is a need for more treatment options that can help fill the significant unmet need that exists for patients today. We are excited by BridgeBio’s recent NDA acceptance from the FDA, which we hope moves us one step closer to having acoramidis available as a treatment for the ATTR-CM community.”

References

1. BridgeBio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). BridgeBio. News release. February 5, 2024. Accessed February 7, 2024. https://bridgebio.com/news/bridgebio-pharma-announces-u-s-food-and-drug-administration-fda-acceptance-of-new-drug-application-nda-for-acoramidis-for-the-treatment-of-patients-with-transthyretin-amyloid-cardiomyopathy-attr/

2. Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM)[Updated 2023 Apr 27]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK574531

3. BridgeBio Pharma Announces US Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM). BridgeBio. News release. July 17, 2023. Accessed February 7, 2024. https://bridgebio.com/news/bridgebio-announces-consistently-positive-results-from-phase-3-attribute-cm-study-of-acoramidis-for-patients-with-transthyretin-amyloid-cardiomyopathy-attr-cm/

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