- If approved, somatrogon will serve as a
once-weekly treatment option –
Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK)
announced today that the US Food and Drug Administration (FDA) has
accepted for filing the initial Biologics License Application (BLA)
for somatrogon, a long-acting human growth hormone that is intended
to be administered once-weekly for the treatment of pediatric
patients with growth hormone deficiency (GHD).
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The target Prescription Drug User Fee Act (PDUFA) action date
for decision by the FDA is in October 2021. Somatrogon is an
investigational new biologic product that is glycosylated and
comprises the amino acid sequence of human growth hormone and one
copy of the C-terminal peptide (CTP) from the beta chain of human
chorionic gonadotropin (hCG) at the N-terminus and two copies of
CTP (in tandem) at the C-terminus. The glycosylation and CTP
domains account for the half-life of the molecule.
“The FDA’s filing acceptance is an encouraging step closer to
our goal of providing a long-acting, once-weekly therapy for
pediatric patients living with GHD. If approved, somatrogon could
help reduce the burden of daily growth hormone injections on
children, their loved ones, and caregivers,” said Brenda
Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer
Global Product Development. “For 35 years, Pfizer has been
committed to improving the outcomes of patients living with GHD,
and somatrogon is another example of how we are working to
positively impact quality of life and treatment compliance to help
ensure those patients can reach their full potential.”
The submission is supported by the results of a global Phase 3
trial evaluating the safety and efficacy of somatrogon administered
once weekly to pediatric patients with GHD. This study met its
primary endpoint of non-inferiority compared to GENOTROPIN®
(somatropin) for injection administered once daily, as measured by
annual height velocity at 12 months. The top-line results from the
study demonstrated 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 (-0.39, 1.05). In addition, change in
height standard deviation scores at 6 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 6 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 the heights of their age and gender matched peers.
Somatrogon was generally well tolerated in the study and
comparable to that of somatropin administered once-daily with
respect to the types, numbers and severity of the adverse events
observed between the treatment arms.
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 pediatric patients, treatment-naïve
children with growth hormone deficiency who were randomized 1:1
into two arms: somatrogon administered at a dose of 0.66 mg/kg body
weight once-weekly vs GENOTROPIN® (somatropin) administered at a
dose of 0.034 mg/kg body weight once daily. The primary endpoint of
the trial was height velocity at 12 months. Secondary endpoints
included change in height standard deviation at 6 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 an investigational biologic product that is
glycosylated and comprises the amino acid sequence of human growth
hormone and one copy of the C-terminal peptide (CTP) from the beta
chain of human chorionic gonadotropin (hCG) at the N-terminus and
two copies of CTP (in tandem) at the C-terminus. The glycosylation
and CTP domains account for the half-life of the molecule.
Somatrogon has 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® (somatropin)
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.
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PFIZER DISCLOSURE NOTICE:
The information contained in this release is as of January 4,
2021. 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 an
investigational growth hormone deficiency therapy, somatrogon,
including a potential indication in the U.S. for once-weekly
treatment of pediatric patients with growth hormone deficiency,
including its 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 additional
jurisdictions for somatrogon for the treatment of pediatric
patients with growth hormone deficiency or in any jurisdictions for
any other potential indications for somatrogon; whether and when
the FDA may approve the BLA for somatrogon for the treatment of
pediatric patients with growth hormone deficiency and whether and
when regulatory authorities in any jurisdictions may approve any
such other 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 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; uncertainties regarding the impact of COVID-19 on
Pfizer’s or OPKO’s business, operations and financial results; 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, 2019 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" and “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.
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version on businesswire.com: https://www.businesswire.com/news/home/20210104005200/en/
Media Contact: Steve Danehy 212-733-1538
Steven.Danehy@pfizer.com
Investor Contact: Chuck Triano 212-733-3901
Charles.E.Triano@pfizer.com
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