New Drug Application Submitted to PMDA in Japan for Somatrogon, to Treat Pediatric Patients with Growth Hormone Deficiency
January 28 2021 - 8:00AM
OPKO Health, Inc. (NASDAQ: OPK) announced today
that its partner, Pfizer Japan Inc., submitted a New Drug
Application (NDA) to the Ministry of Health, Labour, and Welfare in
Japan 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).
This submission is based on the results of the
Japan Phase 3 and global Phase 3 clinical studies, conducted in
subjects with pediatric GHD, in which the efficacy and safety of
somatrogon administered once weekly were compared with GENOTROPIN®
(somatropin), a recombinant human growth hormone for injection,
administered once daily. In both studies, somatrogon showed
comparable efficacy to GENOTROPIN for the primary endpoint of
annual height velocity at 12 months of treatment. Somatrogon was
generally well tolerated in both studies, with comparable safety to
that of GENOTROPIN administered once-daily with respect to the
types, numbers and severity of the adverse events observed between
the treatment arms.
In 2014, Pfizer Inc. and OPKO Health, Inc. entered
into a worldwide agreement for the development and
commercialization of somatrogon. Under the agreement, OPKO is
responsible for conducting the clinical program, and Pfizer is
responsible for registering and commercializing the product.
About the Japan Phase 3 StudyThe
Phase 3 study of somatrogon in 44 treatment-naïve Japanese
pre-pubertal children with pediatric GHD was a 12-month,
open-label, randomized, active-controlled, parallel-group study of
the efficacy and safety of weekly somatrogon compared to
recombinant human growth hormone (r-hGH), GENOTROPIN® (somatropin)
for injection treatment administered once-daily. Eligible patients
were randomized in a 1:1 ratio to receive either once-weekly
somatrogon or GENOTROPIN administered once-daily (reference
therapy, 0.025 mg/kg/day which is equivalent to 0.175 mg/kg/week).
To obtain pharmacokinetic information of three different weekly
doses in Japanese pediatric GHD patients, somatrogon treated
patients received 0.25 mg/kg/week for 2 weeks, followed by 0.48
mg/kg/week for 2 weeks followed by 0.66 mg/kg/week for the
remaining 46 weeks. Somatrogon was administered subcutaneously
using a single patient use, multi-dose, disposable, pre-filled pen,
the same pen used in the global study, while GENOTROPIN was
administered using approved commercial products in Japan.
About SomatrogonSomatrogon 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
DeficiencyGrowth 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 GENOTROPINGENOTROPIN
(somatropin) 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.
Cautionary Statement Regarding
Forward-Looking StatementsThis press release contains
“forward-looking statements,” as that term is defined under the
Private Securities Litigation Reform Act of 1995 (PSLRA), which
statements may be identified by words such as “expects,” “plans,”
“projects,” “will,” “may,” “anticipates,” “believes,” “should,”
“intends,” “estimates,” and other words of similar meaning,
including statements regarding whether the Ministry of Health,
Labour, and Welfare (“Ministry”) may approve the NDA for somatrogon
for the treatment of pediatric patients with growth hormone
deficiency, whether the Ministry will be satisfied with the results
from our clinical study, whether the Ministry will interpret the
clinical trial data in a similar manner to us, whether, if
approved, somatrogon will be commercially successful in Japan for
its approved indication, that the market for somatrogon exists,
that earlier clinical results of effectiveness and safety may not
be reproducible or indicative of future results, as well as other
non-historical statements about our expectations, beliefs or
intentions regarding our business, technologies and products,
strategies or prospects. Many factors could cause our actual
activities or results to differ materially from the activities and
results anticipated in forward-looking statements. These factors
include those described in the OPKO Health, Inc. Annual Reports on
Form 10-K filed and to be filed with the Securities and Exchange
Commission and in its other filings with the Securities and
Exchange Commission. In addition, forward-looking statements may
also be adversely affected by general market factors, competitive
product development, product availability, federal and state
regulations and legislation, the regulatory process for new
products and indications, manufacturing issues that may arise, and
positions and litigation, among other factors. The forward-looking
statements contained in this press release speak only as of the
date the statements were made, and we do not undertake any
obligation to update forward-looking statements. We intend that all
forward-looking statements be subject to the safe-harbor provisions
of the PSLRA.
Contacts:
LHA Investor RelationsYvonne
Briggs, 310-691-7100ybriggs@lhai.comorBruce Voss, 310-691-7100
bvoss@lhai.com
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