BridgeBio Pharma Announces Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
- Category: Small Molecules
- Published on Wednesday, 06 December 2023 10:50
- Hits: 1681
- NDA submission is based on positive results from ATTRibute-CM Phase 3 study, including a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001), on the primary endpoint (a hierarchical analysis prioritizing in order all-cause mortality (ACM), then 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 6-minute walk distance (6MWD))
- In ATTRibute-CM, acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual CVH frequency, as well as improvements for a large proportion of patients on laboratory and functional measures
- ATTRibute-CM results also demonstrated rapid clinical benefit on the composite endpoint of ACM and CVH in patients treated with acoramidis
- Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified
PALO ALTO, CA, USA I December 05, 2023 I BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that it has submitted an NDA for acoramidis to the U.S. FDA for the treatment of ATTR-CM. The application was based on positive results from ATTRibute-CM, the Company’s Phase 3 study designed to evaluate the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR).
“The submission of the acoramidis NDA marks an important milestone in the journey for acoramidis, a novel, near-complete TTR stabilizer. The totality of the evidence consistently point to the potential to provide important clinical benefits over current therapeutic options for patients living with ATTR-CM,” said Jonathan Fox, President and Chief Medical Officer of BridgeBio Cardiorenal. “The opportunity to register acoramidis with the FDA brings us that much closer to making it broadly available as an important treatment option for ATTR-CM in the US and, soon to follow, globally. We extend our deep appreciation to all of the patients who participated in our clinical trials, their families and caregivers, our dedicated investigators and other collaborators, and our BridgeBio team, who all helped make this possible.”
In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001) on the primary endpoint (a hierarchical analysis prioritizing in order: ACM, then frequency of CVH, then change from baseline in NT-proBNP, then change from baseline in 6MWD). Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified.
BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023. The results included an 81% absolute survival rate on treatment and a 0.29 observed mean annual CVH frequency on treatment; additionally, among subjects on acoramidis that completed a Month 30 visit, 45% of subjects improved from baseline in NT-proBNP, 40% of subjects improved from baseline in 6MWD, and 13% of subjects in improved from baseline in New York Heart Association (NYHA) classification. The results also included placebo and acoramidis time-to-first event Kaplan-Meier curves for a composite of ACM and CVH that separated at Month 3 and continued to diverge steadily through Month 30.
The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. The Company intends to submit additional marketing authorization applications to global health authorities in 2024.
BridgeBio is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.