SAN RAFAEL, Calif.,
July 23, 2020 /PRNewswire/
-- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today
that the company submitted a Marketing Authorization Application
(MAA) to the European Medicines Agency (EMA) for vosoritide, an
investigational, once daily injection analog of C-type Natriuretic
Peptide (CNP) for children with achondroplasia, the most common
form of disproportionate short stature in humans. Subject to
completion of EMA's validation check, BioMarin anticipates the
start of the MAA review to commence in August 2020.
The marketing application is based on the outcomes from the
randomized, double-blind, placebo-controlled Phase 3 study
evaluating the efficacy and safety of vosoritide, announced in
December 2019, and further supported by the long-term safety
and efficacy from the ongoing Phase 2 and Phase 3 extension
studies, and extensive natural history data. If approved,
vosoritide would be the first medicine for the treatment of
achondroplasia in Europe.
The company remains on track to submit a New Drug Application
(NDA) to the U.S. Food and Drug Administration (FDA) in the third
quarter of 2020. Vosoritide has Orphan Drug designation from the
FDA and the EMA.
"Years of scientific research have led to this important point
in the development of the potentially first pharmacological
treatment option for children with achondroplasia. We have
worked alongside patient advocacy groups from around the world
throughout the development, and we appreciate the implications of
developing a treatment option for this community, recognizing that
this potential new treatment would offer a choice for families who
have a child with achondroplasia," said Hank Fuchs, M.D., President Worldwide Research
and Development at BioMarin. "Our goal is to provide a
treatment option that addresses the underlying cause of the
condition and over time demonstrate a reduction of complications
that may result from achondroplasia. We respect the depth and
breadth of views among the community about treatment options and
have sought to be scientifically rigorous in providing a robust
data set for regulators to evaluate the safety and efficacy of
vosoritide. We remain grateful to the physicians and families
who have participated in our studies that have helped increase the
scientific understanding of this investigational treatment."
"This is an important milestone bringing the achondroplasia
community one step closer to a potential therapy," said
Klaus Mohnike, Professor of
Paediatrics at Magdeburg University Hospital in Germany and investigator for the vosoritide
clinical program. "Our scientific and medical knowledge
around skeletal dysplasias and achondroplasia in particular
continues to increase, which can help us treat the underlying cause
of the condition and potentially make a meaningful impact on the
lives of children with achondroplasia."
"It is of critical importance that children with achondroplasia
and their families have drug treatment options to advance the
standard of care for this condition where currently none are
available," said Carmen Alonso
Alvarez, Managing Director of Fundacion ALPE
Foundation. "We look forward to expanding treatment options
that can contribute to the improvement of the health and well-being
of children with achondroplasia."
About Achondroplasia
Achondroplasia, the most common form of disproportionate short
stature 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 licensed medicines for achondroplasia.
About BioMarin
BioMarin is a global biotechnology company that develops and
commercializes innovative therapies for serious and
life-threatening rare and ultra-rare genetic diseases. The
Company's portfolio consists of six commercialized products and
multiple clinical and pre-clinical product candidates. For
additional information, please visit www.biomarin.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc. (BioMarin),
including, without limitation, statements about: BioMarin's
vosoritide development program generally and specifically about the
Company's planned submission of an NDA to the FDA in the third
quarter of 2020 for vosoritide and its anticipation that the review
of the MAA for vosoritide by the EMA will begin in August 2020. 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: final analysis of the
Phase 3 data, results and timing of current and planned preclinical
studies and clinical trials of vosoritide; 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 BioMarin's
Securities and Exchange Commission (SEC) filings, including,
without limitation, BioMarin's Quarterly Report on Form 10-Q for
the quarter ended March 31, 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.
Contacts:
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Investors
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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)
455-7558
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(415)
455-7451
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SOURCE BioMarin Pharmaceutical Inc.