Pfizer Inc. (NYSE: PFE) and OPKO Health, Inc. (NASDAQ: OPK)
announced today that the U.S. Food and Drug Administration (FDA)
issued a Complete Response Letter (CRL) for the Biologics License
Application (BLA) for somatrogon. Somatrogon is an investigational
once-weekly long-acting recombinant human growth hormone for the
treatment of growth hormone deficiency (GHD) in pediatric patients.
Pfizer is evaluating the FDA’s comments and will work with the
agency to determine an appropriate path forward.
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“We remain confident in the potential treatment benefits that
somatrogon has to offer patients around the world,” said Brenda
Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer
Global Product Development. “We will work closely with the FDA to
determine the best path forward to bring this important once-weekly
treatment option to pediatric growth hormone deficiency patients
and their families.”
Regulatory applications for somatrogon have been submitted to
several countries around the world for review. Earlier this week,
Japan’s Ministry of Health, Labour and Welfare approved NGENLA®
(somatrogon) Inj. 24 mg Pens and 60mg Pens, for the long-term
treatment of pediatric patients who have growth failure due to an
inadequate secretion of endogenous growth hormone. In 2021, Health
Canada approved NGENLA® for the long-term treatment of pediatric
patients who have GHD, and Australia’s Therapeutic Goods
Administration (TGA) approved NGENLA® for the long-term treatment
of pediatric patients with growth disturbance due to insufficient
secretion of growth hormone. Furthermore, in December 2021, the
Committee for Medicinal Products for Human Use (CHMP) of EMA issued
a positive opinion recommending somatrogon for marketing
authorization in the EU, to treat children and adolescents from 3
years of age with growth disturbance due to insufficient secretion
of growth hormone. A decision from the European Commission (EC) is
expected in early 2022.
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 for GHD.
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 children.1,2 In
children, this disease can be caused by genetic mutations or
acquired after birth.1,3 Because the patient's pituitary gland
secretes inadequate levels of somatropin, the hormone that causes
growth, a child’s height may be affected and puberty may be
delayed.1,3,4 Without treatment, affected children will have
persistent growth attenuation and a very short height in
adulthood.3,4 Children may also experience other problems with
physical health and mental well-being.3,4
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.
About Pfizer: 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 January 21,
2022. 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;
uncertainties regarding the company’s ability to address the
comments in the complete response letter to the satisfaction of the
FDA; whether and when drug applications may be filed in any other
jurisdictions for any potential indication for somatrogon; whether
and when the BLA pending with the FDA for somatrogon for the
treatment of pediatric patients with growth hormone deficiency may
be approved and whether and when regulatory authorities in any
jurisdictions may approve any such other applications that may be
pending or filed (including the application filed in the EU), 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 and 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 Report 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” and “Forward-Looking Information and
Factors That May Affect Future Results”, 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, www.pfizer.com, and www.opko.com.
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.
1 National Organization for Rare Disorders. Growth Hormone
Deficiency.
https://rarediseases.org/rare-diseases/growth-hormone-deficiency/.
Accessed August 24, 2021. 2 Stanley T. Diagnosis of growth hormone
deficiency in childhood. Curr Opin Endocrinol Diabetes Obes.
2012;19(1):47-52. doi:10.1097/MED.0b13e32834ec952. 3 Cerbone M,
Dattani MT. Progression from isolated growth hormone deficiency to
combined pituitary hormone deficiency. Growth Horm IGF Res.
2017;37:19-25. doi:10.1016/j.ghir.2017.10.005. 4 Ergun-Longmire B,
Wajnrajch M. Growth and growth disorders. Feingold KR, Anawalt B,
Boyce A, et al., editors. Endotext [Internet]. South Dartmouth
(MA): MDText.com, Inc.; 2000. Available from:
https://www.ncbi.nlm.nih.gov/books/NBK279142/
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Pfizer Media Relations PfizerMediaRelations@Pfizer.com +1
(212) 733-1226
Pfizer Investor Relations IR@Pfizer.com +1 (212)
733-4848
OPKO / LHA Investor Relations Yvonne Briggs +1 (310)
691-7100 ybriggs@lhai.com or Bruce Voss +1 (310) 691-7100
bvoss@lhai.com
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