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)
05 Fevereiro 2024 - 9:30AM
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 bridgebio.com and follow us
on LinkedIn and Twitter.
BridgeBio Forward-Looking StatementsThis press
release contains forward-looking statements. Statements in this
press release may include statements that are not historical facts
and are considered forward-looking within the meaning of Section
27A of the Securities Act of 1933, as amended (the Securities Act),
and Section 21E of the Securities Exchange Act of 1934, as amended
(the Exchange Act), which are usually identified by the use of
words such as “anticipates,” “believes,” “estimates,” “expects,”
“intends,” “may,” “plans,” “projects,” “seeks,” “should,”
“continue,” “will,” and variations of such words or similar
expressions. We intend these forward-looking statements to be
covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Exchange Act. 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 forward-looking statements. Except as
required by applicable law, we assume no obligation to update
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.
BridgeBio Media Contact:Vikram
Balicontact@bridgebio.com (650)-789-8220
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