Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Kyowa Kirin
International PLC (KKI), a wholly owned subsidiary of Kyowa Hakko
Kirin Co., Ltd. (Kyowa Hakko Kirin), today announced that the
European Medicines Agency (EMA) has accepted for review the
Marketing Authorization Application (MAA) for KRN23 for the
treatment of X-linked hypophosphatemia (XLH). The MAA was filed and
accepted in late 2016, and an opinion from the Committee for
Medicinal Products for Human Use (CHMP) is expected in the second
half of 2017. Ultragenyx and Kyowa Hakko Kirin entered into a
collaboration and license agreement in August 2013 to develop and
commercialize KRN23.
“Based on the positive results from multiple studies in
pediatric and adult patients with XLH, we are pursuing conditional
marketing authorization in order to accelerate access to this
therapy for patients,” said Emil D. Kakkis, M.D., Ph.D., Chief
Executive Officer and President of Ultragenyx.
“X-linked hypophosphatemia is a debilitating condition which
causes long term pain and distress among sufferers and for which
there are no currently approved treatments that target the
underlying cause,” said Dr. Tom Stratford, President and CEO of
KKI. “As part of Kyowa Hakko Kirin, we strive to contribute to the
health and wellbeing of people around the world through advances in
life sciences and technologies. The acceptance of this filing
brings us one step closer to addressing the unmet medical needs of
patients who suffer from X-linked hypophosphatemia.”
About X-Linked Hypophosphatemia (XLH)
XLH is a disorder of phosphate metabolism caused by phosphate
wasting in the urine leading to severe hypophosphatemia. XLH is the
most common heritable form of rickets (the softening and weakening
of bones), that is inherited as an X-linked dominant trait
affecting both males and females. XLH is a distinctive disease
characterized by inadequate mineralization of bone that leads to a
spectrum of abnormalities, including rickets, progressive bowing of
the leg, osteomalacia, bone pain, waddling gait, short stature,
gross motor impairment, muscle weakness, frequent/poorly healing
pseudofractures, spinal stenosis, enthesopathy, and osteoarthritis.
Most pediatric patients and some adult patients are managed using
oral phosphate replacement and active vitamin D (calcitriol)
therapy, which requires multiple divided doses each day and
monitoring for potential risks such as nephrocalcinosis,
hypercalciuria, and hyperparathyroidism.
About KRN23
KRN23 is an investigational recombinant fully human monoclonal
IgG1 antibody, discovered by Kyowa Hakko Kirin, against the
phosphaturic hormone fibroblast growth factor 23 (FGF23). It is
being developed by Ultragenyx and Kyowa Hakko Kirin to treat XLH
and TIO, diseases characterized by excess activity of FGF23. FGF23
is a hormone that reduces serum levels of phosphorus and active
vitamin D by regulating phosphate excretion and active vitamin D
production by the kidney. Phosphate wasting in XLH and TIO is
caused by excessive levels and activity of FGF23. KRN23 is designed
to bind to, and thereby inhibit, the excessive biological activity
of FGF23. By blocking excess FGF23 in patients with XLH and TIO,
KRN23 is intended to increase phosphate reabsorption from the
kidney and increase the production of vitamin D, which enhances
intestinal absorption of phosphate and calcium.
A Phase 3 program studying KRN23 in adults and Phase 2 and Phase
3 studies in pediatric patients with XLH are ongoing. KRN23 is also
being developed for tumor-induced osteomalacia (TIO), a disease
characterized by typically benign tumors that produce excess levels
of FGF23, which can lead to severe osteomalacia, fractures, bone
and muscle pain, and muscle weakness.
Ultragenyx and Kyowa Hakko Kirin plan to submit a biologics
license application (BLA) to the U.S. FDA for KRN23 in the second
half of 2017. The companies continue to discuss the details of the
planned submission with FDA.
About Ultragenyx
Ultragenyx is a clinical-stage biopharmaceutical company
committed to bringing to market novel products for the treatment of
rare and ultra-rare diseases, with a focus on serious, debilitating
genetic diseases. Founded in 2010, the company has rapidly built a
diverse portfolio of product candidates with the potential to
address diseases for which the unmet medical need is high, the
biology for treatment is clear, and for which there are no approved
therapies.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx's strategy is predicated upon time and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's
website at www.ultragenyx.com.
About Kyowa Kirin
Kyowa Kirin International PLC (KKI) is a subsidiary of Kyowa
Hakko Kirin and is a rapidly growing specialty pharmaceutical
company engaged in the development and commercialization of
prescription medicines for the treatment of unmet therapeutic needs
in Europe and the United States. KKI is headquartered in
Scotland.
Kyowa Hakko Kirin Co., Ltd. is a research-based life sciences
company, with special strengths in biotechnologies. In the core
therapeutic areas of oncology, nephrology and immunology/ allergy,
Kyowa Hakko Kirin leverages leading-edge biotechnologies centered
on antibody technologies, to continually discover innovative new
drugs and to develop and market those drugs world-wide. In this
way, the company is working to realize its vision of becoming a
Japan-based global specialty pharmaceutical company that
contributes to the health and wellbeing of people around the
world.
You can learn more about the business at:
www.kyowa-kirin.com.
Ultragenyx Forward-Looking Statements
Except for the historical information contained herein, the
matters set forth in this press release, including statements
regarding Ultragenyx's expectations regarding the timing of release
of additional data for its product candidates, plans to initiate
additional studies for its product candidates and timing regarding
these studies, plans regarding ongoing studies for existing
programs, plans to make regulatory submissions and the timing for
those submissions and the expected timing for an opinion from the
CHMP, are forward-looking statements within the meaning of the
"safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Such forward-looking statements involve
substantial risks and uncertainties that could cause our clinical
development programs, future results, performance or achievements
to differ significantly from those expressed or implied by the
forward-looking statements. Such risks and uncertainties
include, among others, the uncertainties inherent in the clinical
drug development process, including the regulatory approval
process, the timing of our regulatory filings, and other matters
that could affect the success of our drug development programs,
including KRN23. Ultragenyx undertakes no obligation to update or
revise any forward-looking statements. For a further description of
the risks and uncertainties that could cause actual results to
differ from those expressed in these forward-looking statements, as
well as risks relating to the business of the company in general,
see Ultragenyx's Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on November 8, 2016, and its
subsequent periodic reports filed with the Securities and Exchange
Commission.
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Danielle Keatley
+1-844-758-7273
Contact Kyowa Kirin International PLC
Media
Callum Spreng
Spreng Thomson Ltd. (For Kyowa Kirin International PLC)
+44 (0)141 548 5191
Mobile: +44 (0)7803 970103
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