Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical
company focused on the development of novel products for rare and
ultra-rare diseases, Kyowa Hakko Kirin Co., Ltd. (Kyowa Hakko
Kirin) and Kyowa Kirin International PLC (Kyowa Kirin
International) today announce that the U.S. Food and Drug
Administration (FDA) has accepted the Biologics License Application
(BLA) for burosumab to treat pediatric and adult patients with
X-Linked Hypophosphatemia (XLH) and has granted Priority Review
status. Burosumab previously received Breakthrough Therapy
Designation from the FDA for the treatment of XLH in pediatric
patients one year of age and older.
The FDA has granted burosumab Priority Review
status, which is available to drugs that treat a serious condition
and, if approved, would provide a significant improvement in safety
or effectiveness. The Prescription Drug User Fee Act (PDUFA) action
date for the BLA is April 17, 2018. The Agency has not
informed the companies whether an advisory committee meeting to
discuss the application will be needed. The FDA previously
designated burosumab as a drug for a “rare pediatric disease”,
enabling issuance of a priority review voucher if burosumab is
approved.
"XLH is a debilitating disease and there are no
current treatment options that address the underlying cause,” said
Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President
of Ultragenyx. “We are pleased that the FDA has granted priority
review and are looking forward to working with the agency in the
coming months with the goal of bringing this potential new
treatment to patients as quickly as possible.”
“Burosumab is the culmination of Kyowa Hakko
Kirin’s research activity in diseases in which high levels of FGF
23 are a causative factor,” said Mitsuo Satoh, Executive Officer,
Vice President, Head of Research and Development Division of Kyowa
Hakko Kirin. “I believe burosumab has the potential to be an
effective treatment option for patients with conditions with excess
FGF23 such as XLH and tumor induced osteomalacia and we will keep
working to provide this advance in therapy for patients as soon as
possible.”
Dr. Tom Stratford, President and Chief Executive
of Kyowa Kirin International, said, “Burosumab has the potential to
treat both pediatric and adult patients with XLH and we welcome the
fact that the FDA has granted burosumab priority review status,
meaning that sufferers may be able to have access to this treatment
earlier.”
Ultragenyx, Kyowa Hakko Kirin and Kyowa Kirin
International, a wholly owned subsidiary of Kyowa Hakko Kirin, have
been collaborating in the development and commercialization of
burosumab globally, based on the collaboration and license
agreement between Kyowa Hakko Kirin and Ultragenyx.
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.
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 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 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.
Forward-Looking Statements
Except for the historical information contained
herein, the matters set forth in this press release, including
statements regarding Ultragenyx's plans or expectations regarding
future regulatory interactions and the potential timing and success
of filings for regulatory approvals, 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 our 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 the company in general, see
Ultragenyx's Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on July 28, 2017, and its
subsequent periodic reports filed with the Securities and Exchange
Commission.
Contact Ultragenyx Pharmaceutical Inc.Investors & MediaRyan
Martins415-483-8257Contact Kyowa Hakko Kirin Co., Ltd.MediaHiroki
Nakamura+81-3-5205-7205Email: media@kyowa-kirin.co.jp
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