—Findings from sensitivity and post-hoc
analyses were presented during a Late Breaking Clinical Trials
Session at the Heart Failure Society of America 22nd Annual
Scientific Meeting—
—Results of new analyses of all-cause mortality
favored tafamidis across all sub-groups—
—29% and 31% reduction in the risk of death
observed in wild-type and hereditary sub-groups, respectively—
Pfizer Inc. (NYSE:PFE) announced today that additional
sensitivity and post-hoc analyses from the Tafamidis Phase 3
Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) study provide
further detail on the effect of tafamidis across wild-type,
hereditary, and New York Heart Association (NYHA) class sub-groups
of patients with transthyretin amyloid cardiomyopathy (ATTR-CM).1
Tafamidis is the only investigational treatment that has completed
a Phase 3 trial evaluating its safety and efficacy for the
treatment of ATTR-CM.1 ATTR-CM is a rare, fatal, and underdiagnosed
condition associated with progressive heart failure for which there
are currently no approved pharmacologic treatments.2
The findings were presented today during the Late Breaking
Clinical Trials session at the Heart Failure Society of America
22nd Annual Scientific Meeting in Nashville, TN. The primary
results were presented at the ESC Congress 2018 in Munich, Germany
on August 27, 2018 and simultaneously published online in the New
England Journal of Medicine (NEJM).
The broader primary results showed tafamidis significantly
reduced the hierarchical combination of both all-cause mortality
and frequency of cardiovascular-related hospitalizations compared
to placebo over a 30-month period (P=0.0006) in patients with
wild-type and hereditary ATTR-CM.1 A new sensitivity analysis
presented today also demonstrated a significant reduction in the
combination of all-cause mortality and frequency of all-cause
hospitalization compared to placebo over a 30-month period
(P=0.0088).1
In addition, tafamidis reduced the risk of all-cause mortality
across all sub-groups (wild-type, hereditary and NYHA I, II and III
functional class) versus placebo. This included a 29% and 31%
reduction in the risk of death observed in wild-type (HR 0.71; 95%
CI [0.474, 1.052]) and hereditary (HR 0.69; 95% CI [0.408,1.167])
sub-groups, respectively.1 Across wild-type and hereditary
sub-groups, tafamidis consistently reduced the decline in the six
minute walk test distance, a measure of functional capacity, and
aspects of quality of life measured by the Kansas City
Cardiomyopathy Questionnaire – Overall Score, compared with placebo
at Month 30. Tafamidis was also well tolerated, with an observed
safety profile comparable to placebo.1
“These additional insights further support tafamidis as a
potential treatment option for people with wild-type or hereditary
ATTR-CM,” said Brenda Cooperstone MD, Senior Vice President and
Chief Development Officer, Rare Disease, Pfizer Global Product
Development. “We look forward to learning more through further
analyses of this study and continue to work with global regulatory
authorities to bring this medicine to patients.”
“Following statistically significant results from the primary
analysis of the Phase 3 ATTR-ACT trial, we were eager to look
deeper into the efficacy of tafamidis in these key ATTR-CM
sub-populations,” Mathew S. Maurer, MD, Arnold and Arlene Goldstein
Professor of Cardiology, New York-Presbyterian Hospital/ Columbia
University Irving Medical Center, and ATTR-ACT study presenter at
the HFSA Scientific Annual Meeting 2018. “The results of these
additional analyses of the data reinforce the findings presented at
the ESC Congress 2018 and published in the New England Journal of
Medicine a matter of weeks ago, underscoring the importance of
increasing awareness of this potentially deadly condition and
diagnosing patients early in the course of their disease
journey.”
In light of the seriousness of the disease and the lack of
pharmacologic treatment options, Pfizer has established an expanded
access treatment protocol to make tafamidis available to ATTR-CM
patients who may benefit from treatment prior to regulatory
approval. The expanded access treatment protocol is posted on
clinicaltrials.gov (NCT02791230) and additional information about
requesting access may be found at www.pfizercares.com. Access to
these programs may vary by country; physicians may contact their
local Pfizer Medical department for further information. Interested
ATTR-CM patients should contact their local physician to discuss
whether accessing tafamidis may be an appropriate option.
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 US Food and Drug Administration (FDA)
granted tafamidis Fast Track and Breakthrough Therapy designations
for ATTR-CM. Additionally, in March 2018, the Ministry of Labor
Health and Welfare in Japan granted SAKIGAKE designation to
tafamidis for this indication.
About the ATTR-ACT Study1
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 capsules compared to placebo. The study included both
patients with the hereditary (ATTRm) form of the disease, and those
with wild-type (ATTRwt) 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.
The ATTR-ACT study demonstrated tafamidis significantly reduced
all-cause mortality (29.5% vs. 42.9%; hazard ratio = 0.70, 95%
confidence interval [CI] 0.51-0.96, P=0.0259) and
cardiovascular-related hospitalizations (0.48 vs 0.70 annualized
rate; relative risk ratio = 0.68, 95% CI 0.56-0.81, P<0.0001),
compared to placebo.1 This represents a 30% reduction in the risk
of mortality and 32% reduction in the rate of
cardiovascular-related hospitalization. The findings also showed a
consistent directional mortality benefit of tafamidis across all
sub-groups.1
Secondary study endpoints also showed tafamidis reduced the
decline in the six minute walk test distance (P<0.0001), a
measure of functional capacity, and reduced the decline in aspects
of quality of life measured by the Kansas City Cardiomyopathy
Questionnaire – Overall Score (P<0.0001), compared with placebo
at Month 30. Tafamidis was also well tolerated, with an observed
safety profile comparable to placebo.1
For more information on the ATTR-ACT study, go to
www.clinicaltrials.gov.
Tafamidis is an investigational treatment for transthyretin
amyloid cardiomyopathy and is not approved for this indication.
About ATTR-CM
ATTR-CM is a rare, progressive, and underdiagnosed disease
caused by destabilization of a transport protein called
transthyretin, which is composed of 4 identical sub units (a
tetramer).3 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.2,4
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
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a global portfolio of multiple medicines within a number of disease
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metabolic disorders.1
Pfizer Rare Disease combines pioneering science and deep
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We innovate every day leveraging our global footprint to accelerate
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Click here to learn more about our Rare Disease portfolio and
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DISCLOSURE NOTICE: The information contained in this release is
as of September 18, 2018. 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
jurisdictions for tafamidis for the Potential Indication; whether
and when regulatory authorities in any such 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 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.
_____________________1 Data on file. Pfizer Inc. New York, NY.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 Ando Y, Coelho T, Berk JL, et al. Guideline of
transthyretin related hereditary amyloidosis for clinicians.
Orphanet J Rare Dis. 2013;8:31.4 Pfizer Inc. Rare disease.
http://www.pfizer.com/health-and-wellness/health-topics/rare-diseases/areas-of-focus.
Accessed September 13, 2018.
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Pfizer Inc.Media Relations:Neha
Wadhwa212-733-2835Neha.Wadhwa@pfizer.comorInvestors:Chuck
Triano212-733-3901Charles.E.Triano@pfizer.com
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