NOVATO, California,
TOKYO and LONDON, October 10,
2017 /PRNewswire/ --
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 http://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:
http://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.