SAN RAFAEL, Calif.,
Nov. 9, 2020 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced today
that the Company is expanding its clinical program for vosoritide,
an investigational analog of C-type Natriuretic Peptide (CNP), with
two new Phase 2 studies. The first study is sponsored by
BioMarin to investigate the safety of vosoritide in infants with
achondroplasia at risk of life-threatening foramen magnum
compression. The second study is an investigator-initiated
study sponsored by Children's National Hospital in Washington, D.C. to investigate vosoritide in
children with selected genetic forms of short stature, which
together represent addressable patient populations of approximately
275,000.
Phase 2 Study for Children at Risk of Life-Threatening
Foramen Magnum Compression
BioMarin announced today that the Company has enrolled the first
child in a Phase 2 randomized, controlled, open-label clinical
trial with an open-label extension to investigate the safety of
vosoritide in infants with achondroplasia at risk of requiring
cervicomedullary decompression surgery to alleviate compression at
the foramen magnum, the opening in the base of the skull through
which the spinal cord passes. In addition, the study will also
measure a secondary endpoint to evaluate the effect of vosoritide
on growth of the foramen magnum volume through MRI scans.
Foramen magnum compression is the foremost life-threatening
complications of achondroplasia in infants. This study investigates
the safety of vosoritide in infants aged 0 -1 years of age with
achondroplasia who have evidence of foramen magnum compression
at-risk for requiring cervicomedullary decompression surgery. Those
infants are under close observation for the appearance of
neurological signs of progressive foramen magnum compression, and
the current standard of care is palliative with many eventually
requiring surgery. The study aims to enroll approximately 20
infants, who will be randomized to either current standard of care
plus vosoritide treatment or current standard of care alone for a
two-year period. After the two-year randomized period,
children in the study would be eligible to receive vosoritide in an
open-label, 3-year additional extension period. The study will
examine the incidence of adverse events between the two
groups, volume MRI measurements of the foramen magnum, skull
and spine, and progression to requiring decompression surgery.
"We continue to expand our clinical program of vosoritide for
the treatment of achondroplasia with the first study specifically
looking at the safety of vosoritide administered to infants at risk
of serious and potentially fatal medical complications resulting
from achondroplasia," said Hank
Fuchs, M.D., President, Worldwide Research and Development
at BioMarin. "We are committed to understanding whether
vosoritide can safely reduce the number of adverse outcomes in this
high-risk population and are pleased to support this study as part
of our commitment to advancing the standard of care for
children affected by achondroplasia."
Investigator-Initiated Phase 2 Study by Children's National
Hospital to Explore Vosoritide in Selected Genetic Forms of Short
Stature
In addition, Children's National Hospital in Washington, D.C. the sponsor of the
investigator-initiated study, and BioMarin announced today that the
first children have been enrolled in a Phase 2 study of the
investigational treatment of vosoritide in children with certain
genetic forms of short stature.
This investigator-initiated study will enroll approximately 35
children with short stature in specific genetic categories and will
follow them for a six-month observation period to obtain baseline
growth velocity, safety profile and quality of life assessment.
Study participants will then be treated with vosoritide for
12 months and will be assessed for safety and improvement in growth
outcomes. The primary endpoints include incidence of
treatment emergent adverse events, change from baseline in
annualized growth velocity and change from baseline in height
Z-score.
"Many patients who present with short stature likely have
genetic mutations in genes involved in growth plate
physiology. Those patients with selected causes of short
stature that interact with the CNP pathway may benefit from
treatment with vosoritide, which directly targets the growth
plate," said Andrew Dauber, M.D.,
MMSc, Chief of Endocrinology at Children's National Hospital,
Washington D.C. and lead
investigator of this clinical study. "In this study, our goal
is to understand if vosoritide may be a safe and effective
treatment option for certain genetically defined short stature
syndromes."
"We are pleased to support the clinical research by Dr. Dauber
at Children's National Hospital. We applaud his efforts to
study vosoritide for specific types of short stature beyond
achondroplasia," Fuchs added. "Vosoritide is currently under
development for the treatment of achondroplasia, the most common
form of skeletal dysplasia. We have a good foundation of
safety data to help inform a clinical study in other potential
indications. We look forward to supporting this research to
understand if the CNP pathway represents a target in forms of short
stature with specific genetic mutations, where Dr. Dauber's study
is the first step to advance science in the genetic short stature
space."
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia in
humans, is characterized by slowing of endochondral ossification,
which results in disproportionate short stature and disordered
architecture in the long bones, spine, face and base of the
skull. This condition is caused by a mutation in the
fibroblast growth factor receptor 3 gene (FGFR3), a negative
regulator of bone growth. Beyond disproportionate short stature,
people with achondroplasia can experience serious health
complications, including foramen magnum compression, sleep apnea,
bowed legs, mid-face hypoplasia, permanent sway of the lower back,
spinal stenosis and recurrent ear infections. Some of these
complications can result in the need for invasive surgeries such as
spinal cord decompression and straightening of bowed legs. In
addition, studies show increased mortality at every age.
More than 80% of children with achondroplasia have parents of
average stature and have the condition as the result of a
spontaneous gene mutation. The worldwide incidence rate of
achondroplasia is about one in 25,000 live births. Vosoritide
is being tested in children whose growth plates are still "open",
typically those under 18 years of age. This is approximately
25% of people with achondroplasia. In the
U.S., Europe, Latin America, the Middle East, and
most of Asia Pacific, there are currently no approved
medicines for achondroplasia.
About Genetic Forms of Short Stature
Short stature can be caused by a number of genetic etiologies,
many of which directly affect the growth plate. The
CNP/NPR2 pathway is central to growth
of the chondrocyte, therefore patients with selected genetic causes
of short stature that interact with this pathway could benefit from
treatment with vosoritide, which directly targets the growth plate.
This study specifically focuses on five different genes related to
the CNP/NPR2 pathway, including
patients with deficiency in CNP itself or defects in its receptor
encoded by the NPR2 gene; patients
with hypochondroplasia; patients with defects in the SHOX gene; and
patients with a Rasopathy (a group of genetic disorders that
includes Noonan Syndrome and Neurofibromatosis type 1 amongst other
disorders). Collectively, these categories of genetic short
stature affect approximately 275,000 of the addressable patient
population globally based on incidence rate, diagnosis rate, and
age under 18.
About Children's National Hospital
Children's National Hospital, based in Washington, D.C., celebrates 150 years of
pediatric care, research and commitment to community. Volunteers
opened the hospital in 1870 with 12 beds for children displaced
after the Civil War. Today, 150 years stronger, it is among the
nation's top 10 children's hospitals. It is ranked No. 1 for
newborn care for the fourth straight year and ranked in the
top 10 for endocrinology by U.S. News & World Report.
Children's National is transforming pediatric medicine for all
children. In 2020, construction will be complete on the Children's
National Research & Innovation Campus, the first in the nation
dedicated to pediatric research. Children's National has been
designated twice as a Magnet® hospital, demonstrating
the highest standards of nursing and patient care delivery. This
pediatric academic health system offers expert care through a
convenient, community-based primary care network and specialty
outpatient centers in the D.C., metropolitan area, including the
Maryland and Northern Virginia suburbs. Children's National
is home to the Children's National Research Institute and
Sheikh Zayed Institute for Pediatric Surgical
Innovation and is the nation's seventh-highest NIH-funded
children's hospital. It is recognized for its expertise and
innovation in pediatric care and as a strong voice for children
through advocacy at the local, regional and national
levels.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for people with serious and
life-threatening rare disorders. The company's portfolio consists
of six commercialized products and multiple clinical and
pre-clinical product candidates.
For additional information, please visit www.BMRN.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward-Looking Statement for BioMarin
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc. (BioMarin),
including, without limitation, statements about: the development of
BioMarin's vosoritide program generally, whether vosoritide can
reduce the number of adverse outcomes in this high-risk population,
the potential benefits and possible new indications of vosoritide
for children with short stature that is genetically defined and
involves the CNP/NPR2 pathway, the
timing, design and conduct of the two new Phase 2 studies and the
expected results of such studies. These forward-looking statements
are predictions and involve risks and uncertainties such that
actual results may differ materially from these statements. These
risks and uncertainties include, among others: results and timing
of current and planned preclinical studies and clinical trials of
vosoritide; our ability to enroll participants into such clinical
trials, our ability to successfully manufacture vosoritide; the
content and timing of decisions by the U.S. Food and Drug
Administration, the European Commission and other regulatory
authorities concerning vosoritide; and those other risks and
uncertainties detailed from time to time under the caption "Risk
Factors" and elsewhere in the Company's Securities and Exchange
Commission (SEC) filings, including, without limitation, BioMarin's
Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, and future SEC filings and
reports by BioMarin. BioMarin undertakes no duty or obligation to
update any forward-looking statements contained in this press
release as a result of new information, future events or changes in
its expectations.
BioMarin® is a registered trademark of BioMarin
Pharmaceutical Inc.
Contact:
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Traci
McCarty
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Debra
Charlesworth
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BioMarin
Pharmaceutical Inc.
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BioMarin
Pharmaceutical Inc.
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(415)
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455-7451
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SOURCE BioMarin Pharmaceutical Inc.