New, longer-acting treatment offers option to
reduce the frequency of injections for children with growth hormone
deficiency from daily to once-weekly
Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK)
announced today that the U.S. Food and Drug Administration (FDA)
has approved NGENLA (somatrogon-ghla), a once-weekly, human growth
hormone analog indicated for treatment of pediatric patients aged
three years and older who have growth failure due to inadequate
secretion of endogenous growth hormone. NGENLA is expected to
become available for U.S. prescribing in August 2023.
Growth hormone deficiency (GHD) is a rare disease characterized
by the inadequate secretion of the growth hormone somatropin from
the pituitary gland, affecting one in approximately 4,000 to 10,000
children.1,2 Without treatment, children will have persistent
growth attenuation, a very short height in adulthood, and puberty
may be delayed.1,2,3 Children living with GHD may also experience
challenges in relation to their physical health and mental
well-being.1,2,3
“For more than 30 years, Pfizer has been committed to supporting
children and adults living with growth hormone deficiency,
beginning with the delivery of a medicine that has long been a part
of the standard of care,” said Angela Hwang, Chief Commercial
Officer, President, Global Biopharmaceuticals Business, Pfizer. “We
are excited to bring this next-generation treatment to patients in
the United States, continuing our commitment to helping children
living with this rare growth disorder reach their full
potential.”
The FDA approval is supported by results from a multi-center,
randomized, open-label, active-controlled Phase 3 study which
evaluated the safety and efficacy of NGENLA when administered
once-weekly compared to once-daily somatropin. The study met its
primary endpoint of NGENLA non-inferiority compared to somatropin,
as measured by annual height velocity at 12 months. NGENLA was
generally well tolerated in the study and had a safety profile
comparable to somatropin.
“The approval of NGENLA will be significant for children with
growth hormone deficiency in the U.S. It holds potential to reduce
the treatment burden that can come with daily growth hormone
injections,” said Joel Steelman, M.D., Pediatric Endocrinologist,
Cook Children’s Health Care System. “As a new, longer-acting option
that has the ability to reduce treatment frequency from daily to
weekly, NGENLA could become an important treatment option that can
improve adherence for children being treated for growth hormone
deficiency.”
“Throughout our collaboration with Pfizer, we have worked
tirelessly toward our shared goal of helping children living with
growth hormone disease and their families,” said Phillip Frost,
M.D., Chairman and Chief Executive Officer, OPKO Health. “We are
proud of the clinical development program that supported the FDA
approval of NGENLA and are excited about its potential for these
patients and their families as it becomes available in the United
States.”
NGENLA is approved for the treatment of pediatric GHD in more
than 40 markets including Canada, Australia, Japan, and EU Member
States.
The full Prescribing Information can be found here. If it is not
currently available via this link, it will be visible as soon as
possible as we work to finalize the document. Please check back for
the full information shortly.
About NGENLA (somatrogon-ghla) Injection
NGENLA (somatrogon-ghla) is a human growth hormone that works by
replacing the lack of growth hormone in the body. NGENLA is taken
by injection just below the skin, administered via a device that
allows for titration based on patient need. Compared to the growth
hormone GENOTROPIN® (somatropin), its action in the body lasts
longer, enabling weekly injections instead of daily.
In 2014, Pfizer and OPKO entered into a worldwide agreement for
the development and commercialization of NGENLA for the treatment
of GHD. Under the agreement, OPKO is responsible for conducting the
clinical program and Pfizer is responsible for registering and
commercializing NGENLA for GHD.
About the NGENLA Clinical Program The safety and efficacy
of NGENLA (somatrogon-ghla) was demonstrated in a multi-center,
randomized, open-label, active-controlled Phase 3 study (NCT
02968004). The Phase 3 study enrolled and treated 224 pediatric
patients, treatment-naïve children with growth hormone deficiency
who were randomized 1:1 into two arms: NGENLA (somatrogon-ghla)
once-weekly at a dose of 0.66 mg/kg/day vs somatropin, once-daily
at a dose of 0.034 mg/kg/day. The study met its primary endpoint of
NGENLA non-inferiority compared to somatropin, measured by annual
height velocity at 12 months.
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.1,4 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.1,2,5 Without
treatment, affected children will have persistent growth
attenuation and a very short height in adulthood.1,2 Children may
also experience challenges in relation to physical health and
mental well-being.1,2
Important NGENLA (somatrogon-ghla) Safety Information
- Growth hormone should not be used in children after the growth
plates have closed.
- Growth hormone should not be used in children with some types
of eye problems caused by diabetes (diabetic retinopathy).
- Growth hormone should not be used in children who have cancer
or other tumors.
- Growth hormone should not be used in children who are
critically ill because of some types of heart or stomach surgery,
trauma, or breathing (respiratory) problems.
- Growth hormone should not be used in children with Prader-Willi
syndrome who are very overweight or have breathing problems
including sleep apnea.
- NGENLA should not be used by children who have had an allergic
reaction to somatrogon-ghla or any of the ingredients in NGENLA.
Look for prompt medical attention in case of an allergic
reaction.
- Some children have developed diabetes mellitus while taking
growth hormone. Dosages of diabetes medicines may need to be
adjusted during treatment with NGENLA. Children should be watched
carefully if NGENLA 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 raise the likelihood of a new tumor, particularly some benign
(non-cancerous) brain tumors. This likelihood may be higher in
children who were treated with radiation to the brain or head. Your
child’s health care provider will need to check your child for a
return of cancer or a tumor.
- Children treated with growth hormone have had increased
pressure in the brain. If your child has headaches, eye problems,
nausea (feeling like you are going to be sick), or vomiting,
contact your child’s health care provider.
- NGENLA may decrease thyroid hormone levels. Decreased thyroid
hormone levels may change how well NGENLA works. Your child’s
health care provider will do blood tests to check your child’s
hormone levels.
- Children treated with growth hormone should be checked
regularly for low serum cortisol levels and/or the need to increase
the dose of the glucocorticoids they are taking.
- In children experiencing fast growth, curvature of the spine
may develop or worsen. This is also called scoliosis. Children with
scoliosis should be checked regularly to make sure their scoliosis
does not get worse during their growth hormone therapy.
- Use a different area on the body for each injection. This can
help to avoid skin problems such as lumpiness or soreness.
- Growth hormone treatment may cause serious and constant stomach
(abdominal) pain. This could be a sign of pancreatitis. Tell your
child’s health care provider if your child has any new stomach
(abdominal) pain.
- In studies of NGENLA in children with GHD, side effects
included injection site reactions such as pain, swelling, rash,
itching, or bleeding. Other side effects were the common cold,
headache, fever (high temperature), low red blood cells (anemia),
cough, vomiting, decreased thyroid hormone levels, stomach pain,
rash, or throat pain.
- A health care provider will help you with the first injection.
He or she will also train you on how to inject NGENLA.
- Rx only
About GENOTROPIN (somatropin) GENOTROPIN is a man-made,
prescription treatment option. The indications GENOTROPIN is
approved for vary by market. GENOTROPIN is approved for growth
failure due to GHD and adult GHD, Prader-Willi Syndrome, Idiopathic
Short Stature, Turner Syndrome, Small for Gestational Age (with no
catch-up growth), and Chronic Renal Insufficiency. 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 (somatropin) Safety Information
- Somatropin should not be used for growth promotion in pediatric
patients with closed epiphyses.
- Somatropin is contraindicated in patients with active
proliferative or severe nonproliferative diabetic retinopathy.
- Somatropin is contraindicated in patients with active
malignancy. Because growth hormone deficiency may be a sign of
pituitary or other brain tumors, the presence of such tumors should
be ruled out before treatment is initiated. Somatropin should not
be used in patients with any evidence of progression or recurrence
of an underlying intracranial tumor.
- Somatropin in pharmacologic doses should not be used to treat
patients with acute critical illness due to complications from open
heart surgery, abdominal surgery or multiple accidental traumas, or
those patients with acute respiratory failure due to an increased
mortality. The safety of continuing replacement somatropin
treatment for approved uses in patients who develop these illnesses
has not been established.
- Somatropin is contraindicated in patients with Prader-Willi
syndrome who are severely obese or have respiratory
impairment.
- GENOTROPIN is contraindicated in patients with a known
hypersensitivity to somatropin or any of its excipients. Serious
systemic hypersensitivity reactions including anaphylactic
reactions and angioedema have been reported with postmarketing use
of somatropin products. Patients and caregivers should be informed
that such reactions are possible and that prompt medical attention
should be sought if an allergic reaction occurs.
- New-onset Type-2 diabetes mellitus has been reported. Monitor
patients with glucose intolerance closely; dosage of
antihyperglycemic drug may need to be adjusted. Monitor carefully
if somatropin is administered in combination with glucocorticoid
therapy and/or other drugs metabolized by the CP450 pathway.
- In childhood cancer survivors, an increased risk of a second
neoplasm, in particular meningiomas, has been reported in patients
treated with somatropin after their first neoplasm, particularly
those who were treated with cranial radiation. Children with
certain rare genetic causes of short stature have an increased risk
of developing malignancies. Practitioners should thoroughly
consider the risks and benefits of starting somatropin in these
patients and if treatment is initiated, should carefully monitor
these patients for development of neoplasms. Patients should be
monitored carefully for any malignant transformation of skin
lesions.
- Intracranial hypertension (IH) has been reported in a small
number of patients treated with somatropin. If papilledema is
observed during somatropin treatment, treatment should be stopped
and reassessed. Patients with Turner syndrome and Prader-Willi
syndrome may be at increased risk for the development of IH.
- Undiagnosed/untreated hypothyroidism may prevent an optimal
response to somatropin, in particular, the growth response in
children. Patients with Turner syndrome have an inherently
increased risk of developing autoimmune thyroid disease and primary
hypothyroidism. In patients with growth hormone deficiency, central
(secondary) hypothyroidism may first become evident or worsen
during somatropin treatment. Therefore, patients treated with
somatropin should have periodic thyroid function tests, and thyroid
hormone replacement therapy should be initiated or appropriately
adjusted when indicated.
- Patients treated with somatropin who have or are at risk for
pituitary hormone deficiency(s) may be at risk for reduced serum
cortisol levels and/or unmasking central hypoadrenalism and should
be monitored for reduced serum cortisol levels. In addition,
patients treated with glucocorticoid replacement for pre-existing
hypoadrenalism may require an increase in their maintenance or
stress doses following initiation of somatropin treatment and
should be monitored for reduced cortisol levels and/or need for
glucocorticoid dose increases.
- Progression of scoliosis can occur in patients who experience
rapid growth. Patients with scoliosis should be monitored for
manifestation or progression during somatropin therapy.
- Slipped capital femoral epiphyses may occur more frequently in
patients with endocrine disorders (including GHD and Turner
syndrome) or in patients undergoing rapid growth. Any pediatric
patient with the onset of a limp or complaints of hip or knee pain
during somatropin therapy should be carefully evaluated.
- Somatropin should be used during pregnancy only if clearly
needed and with caution in nursing mothers because it is not known
whether somatropin is excreted in human milk.
- Subcutaneous injection of somatropin at the same site
repeatedly may result in tissue atrophy. This can be avoided by
rotating the injection site.
- Cases of pancreatitis have been reported rarely in children and
adults receiving somatropin treatment, with some evidence
supporting a greater risk in children compared with adults.
Published literature indicates that girls who have Turner syndrome
may be at greater risk than other somatropin-treated children.
Pancreatitis should be considered in any somatropin-treated
patient, especially a child, who develops persistent severe
abdominal pain.
- In clinical trials with GENOTROPIN in pediatric GHD patients,
the following events were reported infrequently: injection site
reactions, including pain or burning associated with the injection,
fibrosis, nodules, rash, inflammation, pigmentation, or bleeding;
lipoatrophy; headache; hematuria; hypothyroidism; and mild
hyperglycemia.
- In clinical studies of 273 pediatric patients born SGA treated
with GENOTROPIN, the following clinically significant events were
reported: mild transient hyperglycemia; 1 patient with benign
intracranial hypertension; 2 patients with central precocious
puberty; 2 patients with jaw prominence; and several patients with
aggravation of preexisting scoliosis, injection site reactions, and
self-limited progression of pigmented nevi. Anti-hGH antibodies
were not detected in any of the patients treated with
GENOTROPIN.
- Deaths have been reported with the use of a growth hormone in
pediatric PWS patients with severe obesity, history of upper airway
obstruction or sleep apnea, and/or unidentified respiratory
infection. Therefore, all patients with PWS should be evaluated and
monitored for signs of upper airway obstruction, sleep apnea, and
respiratory infections, and have effective weight control.
- In clinical trials with GENOTROPIN in pediatric patients with
PWS, the following drug-related events were reported: edema,
aggressiveness, arthralgia, benign intracranial hypertension, hair
loss, headache, and myalgia.
- Somatropin may increase the occurrence of otitis media in
Turner syndrome patients. In 2 clinical studies with GENOTROPIN in
pediatric patients with Turner syndrome, the most frequently
reported adverse events were respiratory illnesses (influenza,
tonsillitis, otitis, sinusitis), joint pain, and urinary tract
infection. The only treatment-related adverse event that occurred
in more than 1 patient was joint pain.
- In 2 clinical studies with GENOTROPIN in pediatric patients
with ISS, the most commonly encountered adverse events included
upper respiratory tract infections, influenza, tonsillitis,
nasopharyngitis, gastroenteritis, headaches, increased appetite,
pyrexia, fracture, altered mood, and arthralgia.
- In clinical trials with GENOTROPIN in adults with GHD, the
majority of side effects were symptoms of fluid retention,
including peripheral swelling/edema, arthralgia, pain and stiffness
of the extremities, myalgia, paresthesia, and hypoesthesia.
Generally, these were transient and dose-dependent.
- In women on oral estrogen replacement, a larger dose of
somatropin may be required to achieve the defined treatment
goal.
- Elderly patients may be more sensitive to the action of
somatropin, and therefore may be more prone to develop adverse
reactions.
- The cartridges of GENOTROPIN contain m-Cresol and should not be
used by patients with a known sensitivity to this
preservative.
- Subcutaneous injection of somatropin at the same site
repeatedly may result in tissue atrophy. This can be avoided by
rotating the injection site.
- Health care providers should supervise the first injection and
provide appropriate training and instruction for the proper use of
all devices for GENOTROPIN.
- Rx only
For the full Prescribing Information for GENOTROPIN, please
visit http://labeling.pfizer.com/ShowLabeling.aspx?id=577.
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
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in the discovery, development, and manufacture of health care
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Pfizer colleagues work across developed and emerging markets to
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About OPKO Health 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 www.opko.com.
DISCLOSURE NOTICE: The information contained in this release is
as of June 28, 2023. 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 NGENLA
(somatrogon-ghla) injection and the U.S. FDA approval to treat
pediatric patients aged three years and older with growth failure
due to inadequate secretion of endogenous growth hormone, 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, uncertainties regarding
the commercial success of NGENLA; the uncertainties inherent in
research and development, including the ability to meet anticipated
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
NGENLA injection for the treatment of pediatric patients with
growth hormone deficiency or in any jurisdictions for any other
potential indications for NGENLA injection; whether and when
regulatory authorities in any jurisdictions may approve any
applications that may be pending or filed for NGENLA, 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
NGENLA injection 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 NGENLA injection; uncertainties regarding
the impact of COVID-19 on Pfizer’s and OPKO’s respective 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, 2022 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
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 www.pfizer.com in the case of Pfizer, and
www.opko.com in the case of OPKO.
1 National Organization for Rare Disorders. Growth Hormone
Deficiency.
https://rarediseases.org/rare-diseases/growth-hormone-deficiency/.
Accessed February 22, 2023. 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 Brod, M,
Højbjerre, L, Alolga, SL, Beck, JF, Wilkinson, L, Rasmussen, MH.
Understanding treatment burden for children treated for growth
hormone deficiency. The Patient-Patient-Centered Outcomes Research.
2017;10(5):653-666. 4 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. 5 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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OPKO: LHA Investor Relations Yvonne Briggs
310-691-7100 ybriggs@lhai.com
Bruce Voss 310-691-7100 bvoss@lhai.com
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