—FDA grants a Priority Review based on Phase 3
ATTR-ACT study findings in ATTR-CM—
Pfizer Inc. (NYSE: PFE) announced today that the US Food and
Drug Administration (FDA) accepted for filing the company’s New
Drug Applications (NDAs) for tafamidis for the treatment of
transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer has
submitted two NDAs based on two forms of tafamidis: meglumine salt
and free acid. Tafamidis is the only product to complete a Phase 3
trial evaluating its efficacy, safety, and tolerability in patients
with ATTR-CM, a rare, fatal, and underdiagnosed condition.1,2
The tafamidis meglumine form (20 mg capsule) has been granted
Priority Review. The FDA grants Priority Review to medicines that
may offer significant advances in treatment or may provide a
treatment where no adequate therapy exists. The target Prescription
Drug User Fee Act (PDUFA) action date for a decision by the FDA is
in July 2019.
The tafamidis free acid form (61 mg capsule) will be under
Standard Review. This form is bioequivalent to the 80 mg tafamidis
meglumine dose, which was administered as four 20 mg capsules in
the pivotal trial; it was developed for patient convenience to
enable a single capsule for daily administration. The target PDUFA
action date for a decision by the FDA is in November 2019.
“The diagnosis of ATTR-CM is often delayed, primarily because
disease awareness is low and patients often present with symptoms
similar to more common causes of heart failure. In fact, we believe
less than one percent of patients living with this disease are
currently diagnosed,” said Brenda Cooperstone MD, Senior Vice
President and Chief Development Officer, Rare Disease, Pfizer
Global Product Development. “The FDA’s filing acceptance is an
encouraging step toward our goal of further raising awareness and
providing a treatment option for ATTR-CM patients who are in
desperate need of an approved pharmacologic therapy. We look
forward to working with the FDA to bring the first treatment for
this deadly disease to patients.”
The submission is based on findings from the pivotal Phase 3
Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study, which
evaluated the efficacy, safety, and tolerability of tafamidis
meglumine compared to placebo for the treatment of patients with
ATTR-CM. In the primary analysis of the study, tafamidis met the
primary endpoint, demonstrating a significant reduction in the
hierarchical combination of all-cause mortality and frequency of
cardiovascular-related hospitalizations compared to placebo over a
30-month period in patients with wild-type or hereditary ATTR-CM
(P=0.0006). Tafamidis was well tolerated, with an observed safety
profile comparable to placebo.3 The primary results were presented
in a Hot Line session at the ESC Congress 2018 in Munich, Germany,
and simultaneously published online in the New England Journal of
Medicine (NEJM) in August 2018. Results from additional sub-group
analyses were presented during the Late Breaking Clinical Trials
session at the Heart Failure Society of America 22nd Annual
Scientific Meeting in Nashville, TN, in September 2018. For more
information on the ATTR-ACT trial, go to
www.clinicaltrials.gov.
About Tafamidis3
Tafamidis is an oral, investigational product being evaluated as
a potential treatment for ATTR-CM. Tafamidis is a small molecule
that selectively binds at specific sites on the transthyretin
tetramer to prevent destabilization of the transthyretin transport
protein and formation of amyloid that causes ATTR-CM. Tafamidis is
not approved for any use in the United States.
Tafamidis was granted Orphan Drug Designation for ATTR-CM in
both the EU and US in 2012 and in Japan in 2018. In June 2017 and
May 2018, respectively, the FDA granted tafamidis Fast Track and
Breakthrough Therapy designations for ATTR-CM. In November 2018,
the FDA granted Priority Review designation for the NDA for
tafamidis meglumine. Additionally, in March 2018, the Ministry of
Labor Health and Welfare in Japan granted SAKIGAKE designation to
tafamidis for this indication. Following the SAKIGAKE designation,
a regulatory marketing application for tafamidis for ATTR-CM was
submitted to the Pharmaceuticals and Medical Devices Agency (PMDA)
in November 2018.
About the ATTR-ACT Study3
ATTR-ACT is a Phase 3 international, multicenter, double-blind,
placebo-controlled, randomized, 3-arm clinical study in 441
patients with ATTR-CM that investigated the efficacy, safety, and
tolerability of an oral daily dose of 20 mg or 80 mg tafamidis
meglumine compared to placebo. The study included both patients
with the hereditary form of the disease, and those with wild-type
form, which is not hereditary and may occur as people age. The
primary analysis of the study, which compared a pooled tafamidis
(80 mg and 20 mg) treatment group to placebo, was the hierarchical
combination of all-cause mortality and frequency of
cardiovascular-related hospitalizations over a 30-month period in
patients with transthyretin amyloid cardiomyopathy.
About ATTR-CM
ATTR-CM is a rare and progressive disease caused by
destabilization of a transport protein called transthyretin, which
is composed of four identical subunits (a tetramer). In ATTR-CM,
heart failure occurs when unstable tetramers dissociate, resulting
in misfolded proteins that aggregate into amyloid fibrils and
deposit predominantly in the heart.1,2
Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses
and impacts millions of patients worldwide,4 representing an
opportunity to apply our knowledge and expertise to help make a
significant impact on addressing unmet medical needs. The Pfizer
focus on rare disease builds on more than two decades of
experience, a dedicated research unit focusing on rare disease, and
a global portfolio of multiple medicines within a number of disease
areas of focus, including hematology, neuroscience, and inherited
metabolic disorders.3
Pfizer Rare Disease combines pioneering science and deep
understanding of how diseases work with insights from innovative
strategic collaborations with academic researchers, patients, and
other companies to deliver transformative treatments and solutions.
We innovate every day leveraging our global footprint to accelerate
the development and delivery of groundbreaking medicines and the
hope of cures.
Click here to learn more about our Rare Disease portfolio and
how we empower patients, engage communities in our clinical
development programs, and support programs that heighten disease
awareness.
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well as many of the world's best-known consumer health care
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DISCLOSURE NOTICE: The information contained in this release is
as of January 14, 2019. Pfizer assumes no obligation to update
forward-looking statements contained in this release as the result
of new information or future events or developments.
This release contains forward-looking information about a
potential indication for tafamidis for the treatment of
transthyretin amyloid cardiomyopathy (the “Potential Indication”)
and Pfizer’s rare disease portfolio, including their potential
benefits, that involves substantial risks and uncertainties that
could cause actual results to differ materially from those
expressed or implied by such statements. Risks and uncertainties
include, among other things, the uncertainties inherent in research
and development, including the ability to meet anticipated clinical
trial commencement and completion dates and regulatory submission
dates, as well as the possibility of unfavorable clinical trial
results, including unfavorable new clinical data and additional
analyses of existing clinical data; the risk that clinical trial
data are subject to differing interpretations, and, even when we
view data as sufficient to support the safety and/or effectiveness
of a product candidate, regulatory authorities may not share our
views and may require additional data or may deny approval
altogether; whether regulatory authorities will be satisfied with
the design of and results from our clinical studies; whether and
when any new or supplemental drug applications may be filed in any
other jurisdictions for tafamidis for the Potential Indication;
whether and when the FDA and the PMDA may approve the pending
applications for tafamidis for the Potential Indication and whether
and when regulatory authorities in any such other jurisdictions
where applications for tafamidis may be pending (including the
application pending with the FDA for the potential treatment of
transthyretin familial amyloid polyneuropathy, for which the
company received a complete response letter in 2012) or filed for
the Potential Indication or any other potential indications for
tafamidis may approve any such applications, which will depend on
the assessment by such regulatory authority of the benefit-risk
profile suggested by the totality of the efficacy and safety
information submitted, and, if approved, whether tafamidis will be
commercially successful; decisions by regulatory authorities
regarding labeling and other matters that could affect the
availability or commercial potential of tafamidis, including for
the Potential Indication; and competitive developments.
A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2017 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors” and
“Forward-Looking Information and Factors That May Affect Future
Results”, as well as in its subsequent reports on Form 8-K, all of
which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.pfizer.com.
References
1 Maurer MS, Elliott P, Merlini G, et. al. Design and rationale
of the phase 3 ATTR-ACT clinical trial (tafamidis in transthyretin
cardiomyopathy clinical trial). Circ Heart Fail. 2017;10:1-7.2
Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related
amyloidoses and the heart: a clinical overview. Nat Rev Cardiol.
2010;7:398-408.3 Data on file. Pfizer Inc. New York, NY.4 Pfizer
Inc. Rare disease.
http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus.
Accessed January 11, 2019.
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version on businesswire.com: https://www.businesswire.com/news/home/20190114005391/en/
Media Relations: Neha
Wadhwa212-733-2835Neha.Wadhwa@pfizer.comInvestor Contact: Chuck
Triano212-733-3901Charles.E.Triano@pfizer.com
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