—VYNDAQEL is the only EC-approved medicine
proven to reduce mortality and frequency of cardiovascular-related
hospitalizations in adults with wild-type or hereditary
ATTR-CM—
—VYNDAQEL is the first approved medicine in the
EU to treat both ATTR-CM and stage 1 symptomatic transthyretin
amyloid polyneuropathy (ATTR-PN)—
Pfizer Inc. (NYSE: PFE) announced today that the European
Commission (EC) has approved VYNDAQEL® (tafamidis), a
once-daily 61 mg oral capsule, for the treatment of wild-type or
hereditary transthyretin amyloidosis in adult patients with
cardiomyopathy (ATTR-CM). VYNDAQEL is the first and only treatment
approved in the European Union (EU) for patients with ATTR-CM.
Prior to this approval, treatment options for patients with ATTR-CM
were restricted to symptom management, and, in rare cases, heart
(or heart and liver) transplant.
“Until today, there were no approved medicines to treat patients
with ATTR-CM in the EU. Today’s approval represents incredible
progress for these patients and reflects our steadfast commitment
to delivering breakthrough medicines to rare disease patients,”
said Paul Levesque, Global President, Pfizer Rare Disease.
“Additionally, with today’s milestone, VYNDAQEL is now the first
treatment to have two formulations approved in the EU to treat
manifestations of transthyretin amyloidosis: one for
cardiomyopathy, and one for stage 1 polyneuropathy.”
ATTR-CM is a rare, underdiagnosed and life-threatening disease
characterized by the buildup of abnormal deposits of misfolded
protein called amyloid in the heart and is defined by restrictive
cardiomyopathy and progressive heart failure. Once diagnosed, the
median life expectancy in patients with ATTR-CM, dependent on
sub-type, is approximately two to 3.5 years.
“Before today, the European transthyretin amyloidosis community
had a dire need for new therapeutic options that can improve
outcomes for patients with cardiomyopathy,” said Thibaud Damy, MD,
coordinator of the French Referral Centers for Cardiac Amyloidosis
and past president of the French Heart Failure and Cardiomyopathy
group, French Society of Cardiology. “VYNDAQEL represents a major
advance for patients, as it can significantly reduce all-cause
mortality and the frequency of cardiovascular-related
hospitalizations in patients with wild-type or hereditary
ATTR-CM.”
The EC approval of VYNDAQEL is based on results from the Phase 3
ATTR-ACT study, the first and only completed global, double-blind,
randomized, placebo-controlled clinical trial to investigate a
pharmacologic therapy for the treatment of ATTR-CM. The study
compared patients who received an oral daily dose of 20 mg or 80 mg
of tafamidis meglumine compared to those who received placebo.
In the primary analysis of the study, VYNDAQEL (tafamidis
meglumine) demonstrated a significant reduction in the hierarchical
combination of all-cause mortality and frequency of
cardiovascular-related hospitalisations compared to placebo over a
30-month period in patients with wild-type or hereditary ATTR-CM
(p=0.0006). Additionally, individual components of the primary
analysis demonstrated a relative reduction in the risk of all-cause
mortality and frequency of cardiovascular-related hospitalization
of 30% (p=0.026) and 32% (p<0.0001), respectively, with VYNDAQEL
versus placebo.
VYNDAQEL also had significant and consistent treatment effects
compared to placebo on functional capacity and health status first
observed at six months and continuing through 30 months.
Specifically, VYNDAQEL reduced the decline in performance on the
six-minute walk test (p<0.0001) and reduced the decline in
health status as measured by the Kansas City Cardiomyopathy
Questionnaire – Overall Summary score (p<0.0001).
VYNDAQEL was well tolerated in this study, with an observed
safety profile comparable to placebo. The frequency of adverse
events in patients treated with VYNDAQEL was generally similar and
comparable to placebo. The approval is also based on findings from
an evaluation of the free acid form of tafamidis 61 mg, which
demonstrated that one 61 mg capsule of tafamidis free acid
corresponds to an 80 mg tafamidis meglumine dose (4 x 20 mg
capsules). The safety of the 61 mg dose was not evaluated in
ATTR-ACT. The tafamidis 61 mg capsule was developed for patient
convenience to enable a single capsule for daily
administration.
In 2011, the tafamidis meglumine 20 mg capsule formulation of
VYNDAQEL was approved in the EU for transthyretin amyloidosis in
adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to
delay peripheral neurologic impairment.
About ATTR Amyloidosis
ATTR amyloidosis is rare, progressive disease characterized by
the abnormal buildup of amyloid deposits composed of misfolded
transthyretin protein in the body’s organs and tissues. ATTR
amyloidosis can impact numerous organs and tissues in the body,
including the peripheral nervous system, and organs such as the
heart, kidneys, gastrointestinal tract and eyes. ATTR-CM and
ATTR-PN are two presentations of the disease.
ATTR-CM affects the heart and leads to restrictive
cardiomyopathy and progressive heart failure. There are two
sub-types of ATTR-CM: hereditary, which is caused by a mutation in
the transthyretin gene and can occur in people as early as their
50s and 60s; or the wild-type form which is associated with aging,
and is thought to be more common, usually affecting men after age
60. Often ATTR-CM is diagnosed only after symptoms have become
severe.
ATTR-PN results from a genetic mutation of the transthyretin
gene causing amyloid fibrils to form in the peripheral and
autonomic nerves. ATTR-PN typically occurs during active adult
years with onset as early as the 30s in some patients, followed by
disease progression that may reach the terminal stage in
approximately 10 years on average from disease onset.
About VYNDAQEL (tafamidis 61 mg) and VYNDAQEL (tafamidis
meglumine 20 mg)
VYNDAQEL (tafamidis 61 mg) and VYNDAQEL (tafamidis meglumine 20
mg) are oral transthyretin stabilizers that selectively bind to
transthyretin, stabilizing the tetramer of the transthyretin
transport protein and slowing the formation of amyloid.
The tafamidis 61 mg capsule corresponds to an 80 mg tafamidis
meglumine dose (4x 20mg capsules) and was developed for patient
convenience to enable a single capsule for daily administration.
VYNDAQEL 61 mg and VYNDAQEL 20 mg are not substitutable on a per
milligram basis.
Tafamidis was granted Orphan Drug Designation for ATTR-CM in
both the EU and US in 2012 and in Japan in 2018. Tafamidis was
approved for the treatment of ATTR-CM in Japan under SAKIGAKE
designation in March 2019, in the United States in May 2019, in the
United Arab Emirates in November 2019, in Brazil in December 2019
and in Canada in January 2020.
VYNDAQEL (tafamidis meglumine) 20 mg was first approved in 2011
in the EU for the treatment of transthyretin amyloid polyneuropathy
(ATTR-PN), in adult patients with stage 1 symptomatic
polyneuropathy to delay peripheral neurologic impairment.
Currently, it is approved for ATTR-PN in over 40 countries,
including Japan, countries in Europe, Brazil, Mexico, Argentina,
Israel, Russia, and South Korea. VYNDAQEL is not approved for
ATTR-PN in the US.
VYNDAQEL (tafamidis), a once-daily 61 mg oral capsule, was
granted marketing authorization for patients with ATTR-CM in the EU
in February 2020.
VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis)
From the U.S. Important Safety Information
Adverse Reactions
In studies in patients with ATTR-CM the frequency of adverse
events in patients treated with VYNDAQEL was similar to
placebo.
Specific Populations
Pregnancy: Based on findings from animal studies,
VYNDAQEL and VYNDAMAX may cause fetal harm when administered to a
pregnant woman.
Lactation: There are no available data on the presence of
tafamidis in human milk, the effect on the breastfed infant, or the
effect on milk production. Tafamidis is present in rat milk. When a
drug is present in animal milk, it is likely the drug will be
present in human milk. Breastfeeding is not recommended during
treatment with VYNDAQEL and VYNDAMAX.
The full prescribing information for VYNDAQEL and VYNDAMAX can
be found here.
Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses
and impacts millions of patients worldwide, 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.
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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DISCLOSURE NOTICE: The information contained in this release is
as of February 18, 2020. 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 VYNDAQEL
(tafamidis), an approval for VYNDAQEL by the European Commission
for the treatment of wild-type or hereditary transthyretin
amyloidosis in adult patients with cardiomyopathy 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, uncertainties regarding the commercial success of VYNDAQEL;
the uncertainties inherent in research and development, including
the ability to meet anticipated clinical endpoints, commencement
and/or completion dates for our clinical trials, regulatory
submission dates, regulatory approval dates and/or launch dates, as
well as the possibility of unfavorable new clinical data and
further analyses of existing clinical data; the risk that clinical
trial data are subject to differing interpretations and assessments
by regulatory authorities; 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 VYNDAQEL; whether and when
regulatory authorities in any other jurisdictions where
applications for VYNDAQEL may be pending or filed for the treatment
of wild-type or hereditary transthyretin amyloidosis or any other
potential indications for VYNDAQEL may approve any such
applications, which will depend on myriad factors, including making
a determination as to whether the product’s benefits outweigh its
known risks and determination of the product’s efficacy, and, if
approved, whether VYNDAQEL will be commercially successful;
decisions by regulatory authorities impacting labeling,
manufacturing processes, safety and/or other matters that could
affect the availability or commercial potential of VYNDAQEL,
including for the treatment of wild-type or hereditary
transthyretin amyloidosis in adult patients with cardiomyopathy
(ATTR-CM); 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, 2018 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.
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Investors: Chuck Triano 212-733-3901
Charles.E.Triano@pfizer.com
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