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)
– Accepted with Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024; FDA not currently planning to hold an advisory committee meeting to discuss application
– Marketing Authorization Application accepted by the European Medicines Agency (EMA) with additional global regulatory submissions planned
– In ATTRibute-CM, acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual cardiovascular-related hospitalization (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 all-cause mortality (ACM) and CVH in patients treated with acoramidis, with time-to-first event Kaplan-Meier curves separating at month 3 and continuing to diverge steadily through Month 30
PALO ALTO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for acoramidis, an investigational drug 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 FDA has set an action date of November 29, 2024 under the PDUFA. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.
“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,” said 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.”
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 N-terminal prohormone of brain natriuretic peptide (NT-proBNP), then change from baseline in 6-minute walk distance (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.
“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. 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,” said Isabelle Lousada, president and CEO of the Amyloidosis Research Consortium, a global nonprofit organization dedicated to advancements in amyloidosis.
The Company also received acceptance of its Marketing Authorization Application with the European Medicines Agency and is preparing for additional global regulatory submissions.
About BridgeBio
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 bridgebiodev.wpengine.com and follow us on LinkedIn and Twitter.
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These forward-looking statements, including statements relating
to the clinical, therapeutic and market potential of our
programs and product candidates, including our clinical
development program for acoramidis for patients with
transthyretin amyloid cardiomyopathy, including statements
relating to the FDA’s planned actions regarding our NDA for
acoramidis for the treatment of ATTR-CM, our preparation and
plans for additional regulatory submissions, the statements
regarding the potential outcome of FDA’s review of the NDA, the
potential of acoramidis to make an important contribution to the
care of patients with ATTR-CM and the commitment and hope to
make acoramidis available as a treatment for ATTR-CM to patients
in the quotes of Dr. Fox and Ms. Lousada, and the timing of
these events, reflect our current views about our plans,
intentions, expectations and strategies, which are based on the
information currently available to us and on assumptions we have
made. Although we believe that our plans, intentions,
expectations, and strategies as reflected in or suggested by
those forward-looking statements are reasonable, we can give no
assurance that the plans, intentions, expectations, or
strategies will be attained or achieved. Furthermore, actual
results may differ materially from those described in the
forward-looking statements and will be affected by a number of
risks, uncertainties and assumptions, including, but not limited
to, initial and ongoing data from our clinical trials not being
indicative of final data, the design and success of ongoing and
planned clinical trials, difficulties with enrollment in our
clinical trials, adverse events that may be encountered in our
clinical trials, the FDA or other regulatory agencies not
agreeing with our regulatory approval strategies, components of
our filings, such as clinical trial designs, conduct and
methodologies, or the sufficiency of data submitted, potential
adverse impacts due to the global COVID-19 pandemic such as
delays in regulatory review, manufacturing and supply chain
interruptions, adverse effects on healthcare systems and
disruption of the global economy, the impacts of current
macroeconomic and geopolitical events, including changing
conditions from the COVID-19 pandemic, hostilities in the Middle
East and Ukraine, increasing rates of inflation and rising
interest rates, on our overall business operations and
expectations, as well as those risks set forth in the Risk
Factors section of our Annual Report on Form 10-K for the year
ended December 31, 2022 and our other filings with the U.S.
Securities and Exchange Commission. Moreover, we operate in a
very competitive and rapidly changing environment in which new
risks emerge from time to time. These forward-looking statements
are based upon the current expectations and beliefs of our
management as of the date of this press release, and are subject
to certain risks and uncertainties that could cause actual
results to differ materially from those described in the
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BridgeBio Media Contact:
Vikram Bali
[email protected]
(650)-789-8220