Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug
Administration (FDA) has approved RAPAMUNE® (sirolimus) for the
treatment of lymphangioleiomyomatosis (LAM), a rare, progressive disease
that affects the lungs, kidneys and the lymphatic system. This is the
first approved treatment that helps stabilize lung function in patients
with LAM.
“Pfizer is proud to gain approval for RAPAMUNE as the first treatment
for patients with LAM, through our work with the FDA, the clinical
investigation team and the LAM Foundation,” said Rory O’Connor, MD,
senior vice president and head of Global Medical Affairs, Global
Innovative Pharmaceuticals Business, Pfizer Inc. “This type of
cooperative effort creates opportunities for innovation in developing
therapies for patients with rare diseases.”
LAM is a rare progressive lung disease that usually affects women during
their childbearing years and can result in abnormal growth of smooth
muscle cells in the lung. Over time, the muscle growth can cause airway
obstructions and limit the delivery of oxygen to the body. Approximately
800 patients in the U.S. are currently diagnosed with LAM, which is a
rare disease and is often fatal. RAPAMUNE is also approved in the U.S.
as an immunosuppressive agent for the prophylaxis of organ rejection in
kidney transplant patients aged 13 years and older.
The FDA approval is based on the results from the Multicenter
International Lymphangioleiomyomatosis Efficacy of Sirolimus or MILES
Trial. The MILES Trial included 89 LAM patients with moderate lung
impairment and showed that those treated with RAPAMUNE for one year
experienced stabilization of lung function measured by forced expiratory
volume in one second (FEV1). The adverse drug reactions observed in this
trial were consistent with the known safety profile for renal transplant
patients receiving RAPAMUNE, with the addition of weight decreased,
which was reported at a greater incidence with RAPAMUNE versus placebo.
Serious adverse events were reported more frequently during the
treatment period in patients receiving RAPAMUNE compared to the placebo
group.
“I am thrilled for families living with LAM,” said Dr. Francis X.
McCormack, director of Pulmonary, Critical Care and Sleep Medicine at
the University of Cincinnati College of Medicine and lead investigator
of the MILES Trial. “The courage of the women who enrolled in the MILES
trial made this possible. I am proud of the 200 investigators,
coordinators and nurses who participated in the MILES trial that enabled
FDA approval, and I sincerely thank all who supported the study.”
Pfizer worked with the FDA, Dr. McCormack and the LAM Foundation to
evaluate RAPAMUNE as a treatment option for LAM in the United States.
The MILES trial was conducted by Dr. McCormack and conducted within the
National Institutes of Health Rare Lung Diseases Consortium. Pfizer
provided study drug and a portion of the funding but had no involvement
in the design or conduct of the study. The LAM Foundation assisted with
the recruitment of patients and logistics for the study.
“This approval is a landmark breakthrough for LAM patients to have
access to this important treatment option," said Susan E. Sherman,
executive director of the LAM Foundation. "It is the result of decades
of work by researchers and women of the LAM community who volunteered
for this pivotal clinical trial.”
About RAPAMUNE
RAPAMUNE is an immunosuppressive agent indicated in the United States
for the prophylaxis of organ rejection in patients aged 13 years and
older receiving kidney transplants. It is prescribed to kidney
transplant patients to help prevent their natural immune system from
rejecting the transplanted kidney. RAPAMUNE is also indicated for the
treatment of patients with LAM. Therapeutic drug monitoring is
recommended for all patients receiving RAPAMUNE. RAPAMUNE has a boxed
warning for infections and certain cancers. Please see the full
prescribing information, including boxed warning and full indications at www.pfizer.com/products/product-detail/rapamune.
Important Safety Information
There is an increased risk of developing infections or certain
cancers, especially lymphoma and skin cancers. RAPAMUNE
(sirolimus) has not been shown to be safe and effective in people who
have had liver or lung transplants. Serious complications and
death may happen in people who take RAPAMUNE after a liver or lung
transplant. You should not take RAPAMUNE if you have had a liver
or lung transplant without talking with your doctor.
Do not take RAPAMUNE if you know you are allergic to sirolimus or any of
the other ingredients in RAPAMUNE. Symptoms of an allergic reaction
include swelling of your face, eyes, or mouth; trouble breathing or
wheezing; throat tightness; chest pain or tightness; feeling dizzy or
faint; and rash or peeling of your skin.
Before taking RAPAMUNE, tell your doctor if you have liver problems,
skin cancer or it runs in your family, high cholesterol or
triglycerides, are breastfeeding or plan to breastfeed, and are pregnant
or plan to become pregnant. Women of childbearing potential should use
effective birth control before therapy, during therapy, and for 12 weeks
after RAPAMUNE therapy has been stopped. RAPAMUNE may interact with
other medicines. Make sure that your doctor is aware of all prescription
and over-the-counter drugs that you are taking, including vitamins,
herbs, and nutritional supplements.
RAPAMUNE may cause swelling in your hands, feet, and in various tissues
of your body. Call your doctor if you have trouble breathing.
RAPAMUNE may cause your wounds to heal slowly or not heal well resulting
in redness, drainage, or opening of the wound.
RAPAMUNE may increase the levels of cholesterol and triglycerides
(lipids or fat) in your blood. Your doctor should do blood tests to
check your lipids during treatment with RAPAMUNE. Your doctor may
recommend treatment if your lipid levels become too high. Your lipid
levels may remain high even if you follow your prescribed treatment plan.
In patients taking RAPAMUNE with cyclosporine, decreased kidney function
has been observed. Your doctor will regularly check your kidney function.
RAPAMUNE may increase protein in your urine. Your doctor may monitor you
for abnormal protein in your urine from time to time.
RAPAMUNE may increase your risk for viral infections. Certain viruses
can live in your body and cause active infections when your immune
system is weak. One of these viruses, BK virus, can affect how your
kidney works and cause your transplanted kidney to fail. A certain virus
can cause a rare serious brain infection called Progressive Multifocal
Leukoencephalopathy causing death or severe disability.
RAPAMUNE may cause potentially life-threatening lung or breathing
problems. Symptoms may include coughing, shortness of breath, or
difficulty breathing.
When RAPAMUNE is taken with cyclosporine or tacrolimus, you may develop
a blood clotting problem resulting in unexplained bleeding or bruising.
The most common side effects of RAPAMUNE in people with renal transplant
include high blood pressure, pain (including stomach and joint pain),
diarrhea, headache, fever, urinary tract infection, low red blood cell
count (anemia), nausea, and low platelet count (cells that help blood to
clot), and high blood sugar (diabetes).
The most common side effects of RAPAMUNE in people with LAM include
mouth sores, diarrhea, stomach pain, nausea, sore throat, acne, chest
pain, upper respiratory tract infection, headache, dizziness, and sore
muscles.
Tell your doctor if you have any side effect that bothers you or that
does not go away.
Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact
millions of patients worldwide, representing an opportunity to apply our
knowledge and expertise to help make a significant impact in addressing
unmet medical needs. The Pfizer focus on rare diseases builds on more
than a decade of experience and a global portfolio of more than 20
medicines approved worldwide that treat rare diseases in the areas of
hematology, neuroscience, inherited metabolic disorders, pulmonology,
and oncology.
Pfizer Inc.: 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, Pfizer has worked to make a difference for all who
rely on us. To learn more, please visit us at www.pfizer.com.
DISCLOSURE NOTICE: The information contained in this release is as of
May 29, 2015. 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 RAPAMUNE and
about an indication in the U.S. for the treatment of
lymphangioleiomyomatosis (the "Indication"), 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 regarding the commercial success of RAPAMUNE
and the 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, 2014 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 SEC
and available at www.sec.gov
and www.pfizer.com.
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