— First and only medicines approved for
patients with either wild-type or hereditary transthyretin amyloid
cardiomyopathy —
Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and
Drug Administration (FDA) has approved both VYNDAQEL®
(tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the
treatment of the cardiomyopathy of wild-type or hereditary
transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce
cardiovascular mortality and cardiovascular-related
hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of
the first-in-class transthyretin stabilizer tafamidis, and the
first and only medicines approved by the FDA to treat ATTR-CM.
Transthyretin amyloid cardiomyopathy is a rare, 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.
Previously, there were no medicines approved to treat ATTR-CM; the
only available options included symptom management, and, in rare
cases, heart (or heart and liver) transplant. It is estimated that
the prevalence of ATTR-CM is approximately 100,000 people in the
U.S. and only one to two percent of those patients are diagnosed
today.
“The approvals of VYNDAQEL and VYNDAMAX are a testament to the
significant research and development investment in our innovative
cardiovascular outcomes trial, ATTR-ACT. We are proud to bring
these medicines to ATTR-CM patients who are in dire need of
treatment,” said Brenda Cooperstone, MD, Senior Vice President and
Chief Development Officer, Rare Disease, Pfizer Global Product
Development. “VYNDAQEL and VYNDAMAX reduce cardiovascular mortality
and the frequency of cardiovascular-related hospital stays in
patients with wild-type or hereditary forms of this rare disease,
giving them a chance for more time with their loved ones.”
“Pfizer's purpose is to deliver breakthrough medicines that
change patients' lives. The approvals of VYNDAQEL and VYNDAMAX
deliver on this promise for patients with ATTR-CM,” said Paul
Levesque, Global President, Rare Disease. “This milestone is a
gamechanger for patients, who until today had no approved medicines
for this rare, debilitating and fatal disease. We will continue to
focus efforts on working with the physician community to increase
awareness and ultimately detection and diagnosis of this
disease.”
The recommended dosage is either VYNDAQEL 80 mg orally
once-daily, taken as four 20 mg capsules, or VYNDAMAX 61 mg orally
once-daily, taken as a single capsule. VYNDAMAX was developed for
patient convenience; VYNDAQEL and VYNDAMAX are not substitutable on
a per milligram basis.
“ATTR-CM is not only fatal, but also significantly
underdiagnosed, with some patients cycling through multiple doctors
and a myriad of tests over a period of years while the disease
progresses,” said Isabelle Lousada, Founder and CEO, Amyloidosis
Research Consortium. “ATTR-CM is a rare disease for which more
education and awareness is needed. The approval of these medicines
represents an important advance for patients; however, it is
equally important that we work as a community to recognize the
critical importance of early diagnosis.”
The FDA approval was based on data from the pivotal Phase 3
Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT),
the first global, double-blind, randomized, placebo-controlled
clinical study to investigate a pharmacological therapy for the
treatment of this disease. In ATTR-ACT, VYNDAQEL significantly
reduced the hierarchical combination of all-cause mortality and
frequency of cardiovascular-related hospitalizations compared to
placebo over a 30-month period (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.
Approximately 80% of total deaths were cardiovascular-related in
both treatment groups. 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 similar to placebo, and similar
proportions of VYNDAQEL-treated patients and placebo-treated
patients discontinued the study drug because of an adverse
event.
Pfizer is committed to helping eligible ATTR-CM patients who
have been prescribed VYNDAQEL or VYNDAMAX gain appropriate access.
Pfizer supports patients by helping them understand their insurance
coverage requirements and can connect eligible patients with
financial assistance resources which may be available including the
Pfizer Patient Assistance Program.*
About ATTR-CMTransthyretin amyloid cardiomyopathy
(ATTR-CM) is a rare and fatal condition that is caused by
destabilization of a transport protein called transthyretin, which
is composed of four identical sub units (a tetramer). When unstable
transthyretin tetramers dissociate, they result in misfolded
proteins that aggregate into amyloid fibrils and deposit in the
heart, causing the heart muscle to become stiff, eventually
resulting in heart failure. There are two sub-types of ATTR-CM:
hereditary, also known as variant, which is caused by a mutation in
the transthyretin gene and can occur in people as early as their
50s and 60s; or with no mutation and associated with aging, known
as the wild-type form, which is thought to be more common and
usually affects men after age 60. Often ATTR-CM is diagnosed only
after symptoms have become severe. Once diagnosed, the median life
expectancy in patients with ATTR-CM, dependent on sub-type, is
approximately two to 3.5 years.
About VYNDAQEL (tafamidis meglumine) and VYNDAMAX
(tafamidis)VYNDAQEL (tafamidis meglumine) and VYNDAMAX
(tafamidis) are oral transthyretin stabilizers that selectively
bind to transthyretin, stabilizing the tetramer of the
transthyretin transport protein and slowing the formation of
amyloid that causes ATTR-CM.
VYNDAMAX 61 mg is a once-daily oral capsule developed for
patient convenience. VYNDAQEL and VYNDAMAX are not substitutable on
a per milligram basis.
VYNDAQEL was granted Orphan Drug Designation for ATTR-CM in both
the EU and U.S. in 2012 and in Japan in 2018. In June 2017 and May
2018, respectively, the FDA granted VYNDAQEL Fast Track and
Breakthrough Therapy designations for ATTR-CM. In November 2018,
the FDA granted Priority Review for the new drug application (NDA)
for VYNDAQEL.
In March 2019, the Ministry of Labor Health and Welfare in Japan
approved VYNDAQEL, under SAKIGAKE designation, for patients with
wild-type and variant forms of ATTR-CM. Regulatory submissions for
the use of VYNDAQEL in patients with ATTR-CM have been submitted to
the European Medicines Agency (EMA) and are under review.
VYNDAQEL was first approved in 2011 in the EU for the treatment
of transthyretin amyloid polyneuropathy (ATTR-PN), in adult
patients with early-stage symptomatic polyneuropathy to delay
peripheral neurologic impairment. ATTR-PN is a neurodegenerative
form of amyloidosis that leads to sensory loss, pain and weakness
in the lower limbs and impairment of the autonomic nervous system,
Currently, it is approved for ATTR-PN in 40 countries, including
Japan, countries in Europe, Brazil, Mexico, Argentina, Israel,
Russia, and South Korea. VYNDAQEL and VYNDAMAX are not approved for
the treatment of ATTR-PN in the U.S.
VYNDAQEL (tafamidis meglumine) and VYNDAMAX
(tafamidis) Important Safety Information
Adverse ReactionsIn studies in patients with ATTR-CM the
frequency of adverse events in patients treated with VYNDAQEL was
similar to placebo.
Specific PopulationsPregnancy: 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 DiseaseRare 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.
Working together for a healthier world®At Pfizer,
we apply science and our global resources to bring therapies to
people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of health care products. Our global
portfolio includes medicines and vaccines as well as many of the
world's best-known consumer health care products. Every day, Pfizer
colleagues work across developed and emerging markets to advance
wellness, prevention, treatments and cures that challenge the most
feared diseases of our time. Consistent with our responsibility as
one of the world's premier innovative biopharmaceutical companies,
we collaborate with health care providers, governments and local
communities to support and expand access to reliable, affordable
health care around the world. For more than 150 years, we have
worked to make a difference for all who rely on us. We routinely
post information that may be important to investors on our website
at www.pfizer.com. In addition, to learn more, please visit us on
www.pfizer.com and follow us on Twitter at @Pfizer and
@Pfizer_News, LinkedIn, YouTube and like us on Facebook at
Facebook.com/Pfizer.
DISCLOSURE NOTICE: The information contained in this release is
as of May 6, 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 Pfizer’s
rare disease portfolio, VYNDAQEL (tafamidis meglumine) and VYNDAMAX
(tafamidis) and approvals in the U.S. for the treatment of adults
with ATTR-CM, 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 and
VYNDAMAX; 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 and VYNDAMAX; whether and when the
pending applications with the EMA and whether and when regulatory
authorities in any other jurisdictions where applications for
VYNDAQEL and VYNDAMAX may be pending or filed 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 and VYNDAMAX 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 and
VYNDAMAX; 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.
*Free medicines from Pfizer are provided through the Pfizer
Patient Assistance Foundation™. The Pfizer Patient Assistance
Foundation is a separate legal entity from Pfizer Inc. with
distinct legal restrictions.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190506005185/en/
Media Relations: Steve
Danehy212-733-1538Steven.Danehy@pfizer.com
Investors: Chuck
Triano212-733-3901Charles.E.Triano@pfizer.com
Pfizer (NYSE:PFE)
Historical Stock Chart
From Apr 2024 to May 2024
Pfizer (NYSE:PFE)
Historical Stock Chart
From May 2023 to May 2024