Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko Kirin), Kyowa Kirin
International PLC (Kyowa Kirin International) and Ultragenyx
Pharmaceutical Inc. (NASDAQ:RARE), today announced that the
Committee for Medicinal Products for Human Use (CHMP), the European
Medicines Agency’s (EMA) scientific committee, has adopted a
Positive Opinion recommending the conditional marketing
authorization of burosumab, an anti-FGF23 human monoclonal
antibody, for the treatment of X-linked hypophosphatemia (XLH) with
radiographic evidence of bone disease in children 1 year of age and
older and adolescents with growing skeletons. XLH is a rare,
genetic, chronic and progressive musculoskeletal disorder. If
approved, burosumab would be the first therapy that addresses the
excess FGF23 activity in XLH.
The CHMP’s opinion is now being referred to the
European Commission (EC), for a final decision on the grant of a
conditional marketing authorization. This decision is expected in
the first quarter of 2018 and will apply to all 28 countries of the
European Union, Norway, Iceland and Liechtenstein. The conditional
authorization requires fulfilment of specific obligations related
to the completion of ongoing clinical studies of burosumab in
pediatric patients.
“Our scientists at Kyowa Hakko Kirin were the
first to discover and succeed in cloning FGF23 and identify the
role of the protein in treating phosphate wasting diseases. This
prompted a robust research programme and clinical development
collaboration with Ultragenyx to develop burosumab, an antibody to
FGF23,” said Mitsuo Satoh, Ph.D., Executive Officer, Vice President
Head of R&D Division of Kyowa Hakko Kirin. “We are pleased to
reach this significant regulatory milestone and will continue our
scientific journey to help address the medical needs of people with
XLH.”
Dr. Tom Stratford, President and Chief Executive
of Kyowa Kirin International, said: “At Kyowa Kirin International
we are fully committed to improving the lives of the many young
people across Europe who are living with XLH. We welcome the CHMP’s
opinion which takes us one step closer to launching burosumab
across Europe as the first targeted treatment with the potential to
address this often painful and debilitating musculoskeletal
condition.”
"The positive CHMP opinion is an important step
in accelerating patient access to this therapy, which could
fundamentally change how this disease is treated,” said Emil D.
Kakkis, M.D., Ph.D., Chief Executive Officer and President of
Ultragenyx.
Kyowa Hakko Kirin, Kyowa Kirin International, a
wholly owned subsidiary of Kyowa Hakko Kirin, and Ultragenyx, have
been collaborating in the development and commercialization of
burosumab globally, based on the collaboration and license
agreement between Kyowa Hakko Kirin and Ultragenyx.
Burosumab Regulatory Status
The EMA’s scientific committee adopted a
Positive Opinion recommending the conditional marketing
authorization of burosumab for the treatment of X-linked
hypophosphatemia (XLH) with radiographic evidence of bone disease
in children 1 year of age and older and adolescents with growing
skeletons. The CHMP’s recommendation is now being referred to the
European Commission (EC), which is expected to render its final
decision in the first quarter of 2018. The EC typically adheres to
the recommendation of the CHMP, but is not obligated to do so.
About X-Linked Hypophosphatemia
(XLH)
XLH is a rare, chronic progressive
musculoskeletal disorder characterised by renal phosphate wasting
caused by excess FGF23 production, and is inherited as an X-linked
dominant trait affecting both males and females. XLH is first seen
in infants and also affects adults.
In children, XLH causes skeletal disease,
leading to lower-extremity deformity and diminished height.
The conventional treatment of XLH consists of
multiple daily doses of phosphate and active vitamin D to
counteract the excess effects of FGF23 but does not correct the
underlying disease.
About burosumab
Burosumab is an investigational recombinant
fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko
Kirin, against the phosphaturic hormone fibroblast growth factor 23
(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. Burosumab is being developed to
treat XLH and tumor-induced osteomalacia (TIO), diseases
characterized by excess levels of FGF23. Phosphate wasting in XLH
and TIO is caused by excessive levels and activity of FGF23.
Burosumab is designed to bind to and thereby inhibit the biological
activity of FGF23. By blocking excess FGF23 in patients with XLH
and TIO, burosumab is intended to increase phosphate reabsorption
from the kidney and increase the production of vitamin D, which
enhances intestinal absorption of phosphate and calcium.
In the United States, the U.S. Food and Drug
Administration (FDA) is currently reviewing the Biologics License
Application for burosumab to treat pediatric and adult patients
with XLH, and has set a Prescription Drug User Fee Act (PDUFA)
action date of April 17, 2018.
A clinical program studying burosumab in adults
and pediatric patients with XLH is ongoing. Burosumab is also being
developed for 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.
About Kyowa Kirin
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.
Kyowa Kirin International PLC is a wholly owned
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. Kyowa
Kirin International is headquartered in Scotland.
You can learn more about the business at:
www.kyowa-kirin.com.
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.
Forward-Looking Statements
Except for the historical information contained
herein, the matters set forth in this press release, including
statements relating to Ultragenyx's expectations regarding future
regulatory interactions, the potential timing and success of
filings for regulatory approvals, potential indications for its
product candidates and plans for its clinical programs and clinical
studies, 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, such as the regulatory approval process, the
timing of regulatory filings, and other matters that could affect
sufficiency of existing cash, cash equivalents and short-term
investments to fund operations and the availability or commercial
potential of our drug candidates. 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 Ultragenyx in general, see Ultragenyx's Quarterly
Report on Form 10-Q filed with the Securities and Exchange
Commission on November 3, 2017, and its subsequent periodic reports
filed with the Securities and Exchange Commission.
Contact Kyowa Hakko Kirin Co. Ltd.MediaHiroki
Nakamura+81-3-5205-7205Email: media@kyowa-kirin.co.jp
Contact Kyowa Kirin International PLCMediaCallum SprengSpreng
Thomson Ltd. (For Kyowa Kirin International PLC)+44 (0)141 548
5191Mobile: +44 (0)7803 970103
Contact Ultragenyx Pharmaceutical Inc.Investors &
MediaDanielle Keatley+1-415-475-6876
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