– 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 European Medicines Agency (EMA) has
validated for review the Marketing Authorization Application (MAA)
for somatrogon, a long-acting recombinant human growth hormone that
is intended to be administered once-weekly for the treatment of
pediatric patients with growth hormone deficiency (GHD). Pfizer
expects a decision from the European Commission in 2022.
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“Today’s announcement is an example of our decades-long
commitment to actively support the pediatric growth hormone
deficiency community through therapeutic options that help children
reach their full potential,” said Brenda Cooperstone, MD, Chief
Development Officer, Rare Disease, Pfizer Global Product
Development. “If approved in the EU, somatrogon will represent an
important advancement, as this long-acting, weekly treatment may
reduce the burden of daily injections on children, their loved
ones, and caregivers. We look forward to continuing to work with
the EMA to evolve the treatment paradigm for pediatric patients
with growth hormone deficiency.”
“We are very appreciative of the children and their families
that participated in the clinical trials, bringing us one step
closer to providing a long-acting treatment option for pediatric
growth hormone deficiency. If somatrogon is approved, we look
forward to positively impacting the quality of life for children
living with GHD,” said Phillip Frost, Chairman and CEO of OPKO.
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. 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.
Separately, in October 2020, Pfizer and OPKO announced findings
from the C0311002 trial, a Phase 3, randomized, multicenter,
open-label, crossover study evaluating somatrogon dosed once-weekly
in children 3 to <18 years of age with GHD, which met its
primary endpoint of improved treatment burden compared to
GENOTROPIN® (somatropin) for injection administered once-daily.
Top-line results from the study demonstrated that treatment with
somatrogon once-weekly improved the mean overall Life Interference
total score after 12 weeks of treatment compared to treatment with
somatropin administered once-daily. In addition, key secondary
endpoints showed an overall benefit in treatment experience with
the somatrogon once-weekly dosing regimen compared to the
somatropin once-daily dosing regimen. These data have also been
submitted to the EMA.
In January 2021, Pfizer and OPKO announced that the US Food and
Drug Administration (FDA) accepted for filing the initial Biologics
License Application (BLA) for somatrogon with a target PDUFA action
date of October 2021. In January 2021, a New Drug Application (NDA)
was submitted to PMDA in Japan for somatrogon.
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 Studies
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.
C0311002 is a Phase 3, randomized, multicenter, open-label,
crossover study assessing subject perception of treatment burden
with use of somatrogon administered once-weekly versus GENOTROPIN®
administered once-daily in children 3 to <18 years of age with
growth hormone deficiency (GHD). The primary objective of the
crossover study, which included 87 randomized and treated subjects
(43 randomized to Sequence 1 [somatropin followed by somatrogon]
and 44 randomized to Sequence 2 [somatrogon followed by
somatropin], was to evaluate the treatment burden of a somatrogon
once-weekly injection schedule and a somatropin once-daily
injection schedule, as assessed by the difference in mean overall
Life Interference total scores after each 12-week treatment
schedule experience.
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 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.
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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DISCLOSURE NOTICE:
The information contained in this release is as of February 26,
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 EU 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 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 European Commission may approve the marketing authorization
application 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 that may be pending or filed (including the
applications pending in the U.S. and Japan), 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, 2020 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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