— First and only medicines approved for patients with either
wild-type or hereditary transthyretin amyloid cardiomyopathy —
NEW YORK–(BUSINESS WIRE)–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-CM
Transthyretin 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 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
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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
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support programs that heighten disease awareness.
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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.
Contacts
Media Relations: Steve Danehy
212-733-1538
Steven.Danehy@pfizer.com
Investors: Chuck Triano
212-733-3901
Charles.E.Triano@pfizer.com