OPKO Health Inc. (NASDAQ: OPK) and Pfizer Inc. (NYSE: PFE)
announced today that the global Phase 3 trial evaluating somatrogon
dosed once-weekly in pre-pubertal children with growth hormone
deficiency (GHD) met its primary endpoint of non-inferiority to
daily GENOTROPIN® (somatropin) for injection, as measured by annual
height velocity at 12 months.
Top-line results from the study demonstrated
that treatment with somatrogon dosed once-weekly in pre-pubertal
children with GHD was non-inferior to somatropin dosed once-daily
with respect to height velocity at 12 months of treatment (the
primary endpoint); the least square mean was higher in the
somatrogon group (10.12 cm/year) than in the somatropin group (9.78
cm/year); the treatment difference (somatrogon – somatropin) in
height velocity (cm/year) was 0.33 with a two-sided 95% confidence
interval of the difference of (-0.39, 1.05). In addition, change in
height standard deviation scores at six and 12 months, key
secondary endpoints, were higher in the somatrogon dosed
once-weekly cohort in comparison to the somatropin dosed once-daily
cohort. Moreover, at six months, change in height velocity, another
key secondary endpoint, was higher in the somatrogon dosed
once-weekly cohort in comparison to the somatropin dosed once-daily
cohort. These common measures of growth are employed in the
clinical setting to measure the potential level of catch-up growth
that subjects may experience relative to heights of age and gender
matched peers.
Somatrogon was generally well tolerated in the
study and comparable to that of somatropin dosed once-daily with
respect to the types, numbers and severity of the adverse events
observed between the treatment arms. Immunogenicity testing and
analysis of additional data are ongoing, and full results of the
study will be submitted for presentation at a future scientific
meeting.
“We are very pleased to announce these favorable
top-line results demonstrating the efficacy of somatrogon dosed
once-weekly in the pediatric patient population with GHD,” said
Phillip Frost, M.D., Chairman and Chief Executive Officer of OPKO.
“Somatrogon is a new molecular entity incorporating OPKO’s
proprietary long-acting technology. We believe somatrogon
represents a significant advance in the treatment of children with
GHD compared to the current standard of one injection per day that
could enhance a patient’s adherence to treatment and quality of
life.”
“We’re encouraged by these data and look forward
to the possibility of bringing this longer-acting therapy to
children. If approved, somatrogon could reduce the daily disease
burden on children and their caregivers, potentially increasing
treatment adherence,” said Brenda Cooperstone, M.D., Chief
Development Officer, Rare Disease, Pfizer Global Product
Development. “At Pfizer, we strive each and every day to advance
treatment options that better serve the needs of patients with rare
diseases.”
In 2014, Pfizer and OPKO entered into a
worldwide agreement for the development and commercialization of
somatrogon for the treatment of GHD. Under the agreement, OPKO is
responsible for conducting the clinical program and Pfizer is
responsible for registering and commercializing the product.
About the Study
The somatrogon Phase 3 trial is a randomized,
open-label, active-controlled study conducted in over 20 countries.
This study enrolled and treated 224 pre-pubertal, treatment-naïve
children with growth hormone deficiency who were randomized 1:1
into two arms: somatrogon dosed once-weekly vs daily GENOTROPIN®
(somatropin) for injection. The primary endpoint of the trial was
height velocity at 12 months. Secondary endpoints included change
in height standard deviation at six and 12 months, safety and
pharmacodynamic measures. Children completing this study had
the opportunity to enroll in a global, open-label, multicenter,
long-term extension study, in which they were able to either
continue receiving or switch to somatrogon. Approximately 95% of
the patients switched into the open-label extension study and
received somatrogon treatment.
About Somatrogon
Somatrogon is a new molecular entity that
contains the natural sequence of growth hormone and one copy of the
C-terminal peptide (CTP) from the beta chain of human chorionic hCG
at the N-terminus and two copies at the C-terminus. The CTPs extend
the half-life of the molecule. Somatrogon received Orphan Drug
designation in the U.S. and the EU for the treatment of children
and adults with growth hormone deficiency.
About Growth Hormone
Deficiency
Growth hormone deficiency is a rare disease
characterized by the inadequate secretion of growth hormone from
the pituitary gland and affects one in approximately 4,000 to
10,000 people. In children, this disease can be caused by genetic
mutations or acquired after birth. Because the patient's pituitary
gland secretes inadequate levels of somatropin, the hormone that
causes growth, his or her height may be affected, and puberty may
be delayed. Without treatment, he or she will have persistent
growth attenuation, a very short height in adulthood, and may
experience other health problems.
About GENOTROPIN®
GENOTROPIN is a man-made, prescription treatment
option, approved in the United States for children who do not make
enough growth hormone on their own, have the genetic condition
called Prader-Willi syndrome (PWS), were born smaller than most
other babies, have the genetic condition called Turner syndrome
(TS) or have idiopathic short stature (ISS). GENOTROPIN is also
approved to treat adults with growth hormone deficiency. GENOTROPIN
is taken by injection just below the skin and is available in a
wide range of devices to fit a range of individual dosing needs.
GENOTROPIN is just like the natural growth hormone that our bodies
make and has an established safety profile.
Important GENOTROPIN® Safety
Information
- Growth hormone should not be used
to increase height in children after the growth plates have
closed.
- Growth hormone should not be used
in patients with diabetes who have certain types of diabetic
retinopathy (eye problems).
- Growth hormone should not be used
in patients with cancer or who are being treated for cancer. Growth
hormone deficiency can be caused by brain tumors. So, the presence
of these brain tumors should be ruled out before treatment is
started. Growth hormone should not be used if it is shown that a
previous brain tumor has come back or is getting larger.
- Growth hormone should not be used
in patients who are critically ill because of surgery, trauma, or
respiratory failure.
- Growth hormone should not be used
in children with Prader-Willi syndrome who are very overweight or
have severe breathing problems.
- GENOTROPIN should not be used by
patients who have had an allergy or bad reaction to somatropin or
any of the other ingredients in GENOTROPIN.
- Some patients have developed
diabetes mellitus while taking GENOTROPIN. Dosage of diabetes
medicines may need to be adjusted during growth hormone treatment.
Patients should be watched carefully if growth hormone is given
along with glucocorticoid therapy and/or other drugs that are
processed by the body in the same way.
- In childhood cancer survivors,
treatment with growth hormone may increase the risk of a new tumor,
particularly certain benign brain tumors. This risk may be higher
in patients who were treated with cranial radiation. Also, patients
and their doctors should check regularly for any skin changes.
- A small number of patients treated
with growth hormone have had increased pressure in the brain. This
can cause headaches and problems with vision. Treatment should be
stopped and reassessed in these patients. Patients with Turner
syndrome and Prader-Willi syndrome may be at higher risk of
developing increased pressure in the brain.
- Thyroid function should be checked
regularly during growth hormone therapy. Thyroid hormone
replacement therapy should be started or adjusted if needed.
- Patients treated with growth
hormone should be checked regularly if they are receiving standard
hormone replacement therapy to treat a lack of more than one
hormone.
- In children experiencing rapid
growth, curvature of the spine may develop or worsen. This is also
called scoliosis. Patients with scoliosis should be checked
regularly to make sure their scoliosis does not get worse during
their growth hormone therapy.
- In children experiencing rapid
growth, limping or hip or knee pain may occur. If a child getting
growth hormone therapy starts to limp or gets hip or knee pain, the
child’s doctor should be notified and the child should be
examined.
- Growth hormone should only be used
during pregnancy if clearly needed. It should be used with caution
in nursing mothers because it is not known whether growth hormone
is passed into human milk.
- Use a different place on the body
each day for growth hormone injections. This can help to prevent
skin problems such as lumpiness or soreness.
- Some cases of pancreatitis
(inflamed pancreas) have been reported rarely in children and
adults receiving growth hormone. There is some evidence that there
is a greater risk of this in children than in adults. Literature
suggests that girls who have Turner syndrome may have a greater
risk of pancreatitis than other children taking growth hormone. In
any child who develops lasting, severe abdominal pain, pancreatitis
should be considered.
- In studies of GENOTROPIN in
children with GHD, side effects included injection site reactions,
such as pain, redness/swelling, inflammation, bleeding, scarring,
lumps, or rash. Other side effects were fat loss, headache, blood
in the urine, low thyroid activity, and mildly increased blood
sugar.
- In studies of GENOTROPIN in
children born SGA, side effects included temporarily elevated blood
sugar, increased pressure in the brain, early puberty, abnormal jaw
growth, injection site reactions, growth of moles, and worsening of
scoliosis (curvature of the spine).
- Deaths have been reported with the
use of growth hormone in children with Prader-Willi syndrome. These
children were extremely overweight, had breathing problems, and/or
lung infection. All patients with Prader-Willi syndrome should be
examined for these problems. They should also establish healthy
weight control.
- In studies of GENOTROPIN in
children with PWS, side effects included fluid retention,
aggressiveness, joint and muscle pain, hair loss, headache, and
increased pressure in the brain.
- Turner syndrome patients taking
growth hormone therapy may be more likely to get ear infections.
This is also called otitis media.
- In studies of GENOTROPIN in
children with Turner syndrome, side effects included flu, throat,
ear, or sinus infection, runny nose, joint pain, and urinary tract
infection.
- In studies of GENOTROPIN in
children with ISS, side effects included respiratory illnesses,
flu, throat infection, inflammation of the nose and throat, stomach
pain, headaches, increased appetite, fever, fracture, mood changes,
and joint pain.
- Women who are taking estrogen by
mouth may take GENOTROPIN. They may need a larger dose of growth
hormone.
- GENOTROPIN may be taken by the
elderly. Elderly patients may be more likely to have side effects
with growth hormone therapy.
- In studies of GENOTROPIN in adults
with GHD, side effects included fluid retention, joint or muscle
pain, stiffness, and changes in sensation. Usually these side
effects did not last long and depended on the dose of GENOTROPIN
being taken.
- GENOTROPIN cartridges contain
m-Cresol and should not be used by patients allergic to it.
- A health care provider will help
you with the first injection. He or she will also train you on how
to inject GENOTROPIN.
You are encouraged to report negative side
effects of prescription drugs to the FDA. Visit
www.fda.gov/medwatch or call 1-800-FDA-1088.
For the full Prescribing Information for GENOTROPIN, please
visit http://labeling.pfizer.com/ShowLabeling.aspx?id=577.
GENOTROPIN Indications and Usage in the
U.S.
GENOTROPIN is a prescription product for the
treatment of growth failure in children:
- Who do not make enough growth
hormone on their own. This condition is called growth hormone
deficiency (GHD).
- With a genetic condition called
Prader-Willi syndrome (PWS). Growth hormone is not right for all
children with PWS. Check with your doctor.
- Who were born smaller than most
other babies born after the same number of weeks of pregnancy. Some
of these babies may not show catch-up growth by age 2 years. This
condition is called small for gestational age (SGA).
- With a genetic condition called
Turner syndrome (TS).
- With idiopathic short stature
(ISS), which means that they are shorter than 98.8% of other
children of the same age and sex; they are growing at a rate that
is not likely to allow them to reach normal adult height, and their
growth plates have not closed. Other causes of short height should
be ruled out. ISS has no known cause.
GENOTROPIN is a prescription product for the
replacement of growth hormone in adults with growth hormone
deficiency (GHD) that started either in childhood or as an adult.
Your doctor should do tests to be sure you have GHD, as
appropriate.
About OPKO Health, Inc.
OPKO is a multinational biopharmaceutical and
diagnostics company that seeks to establish industry-leading
positions in large, rapidly growing markets by leveraging its
discovery, development, and commercialization expertise and novel
and proprietary technologies. For more information, visit
http://www.OPKO.com. 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 rare hematologic, neurologic, cardiac 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.
Pfizer Inc.: Breakthroughs that change
patients’ lives
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, including innovative medicines
and vaccines. 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 October 21, 2019.
Pfizer and OPKO assume 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 product candidate, somatrogon dosed once-weekly
in pre-pubertal children with growth hormone deficiency (or GHD)
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
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 drug
applications may be filed in any jurisdictions for somatrogon;
whether and when any such applications may be approved by
regulatory authorities, 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 somatrogon 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 somatrogon; and competitive developments.
A further description of risks and uncertainties
can be found in Pfizer’s and OPKO’s respective Annual Reports on
Form 10-K for the fiscal year ended December 31, 2018 and in their
respective subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors”, “Forward-Looking
Information and Factors That May Affect Future Results”,
“Cautionary Statement Regarding Forward-Looking Statements”, as
well as in their respective 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, as
applicable, www.pfizer.com and www.OPKO.com.
OPKO Health Contacts:LHA Investor RelationsMiriam
Weber Miller, 212-838-3777MMiller@lhai.comOrBruce Voss,
310-691-7100BVoss@lhai.com |
Pfizer Media Contact:Steve Danehy,
212-733-1538Steven.Danehy@pfizer.comPfizer Investor
Contact:Chuck Triano,
212-733-3901Charles.E.Triano@pfizer.com |
Pfizer (NYSE:PFE)
Gráfico Histórico do Ativo
De Mar 2024 até Abr 2024
Pfizer (NYSE:PFE)
Gráfico Histórico do Ativo
De Abr 2023 até Abr 2024