NOVATO, California and
LONDON, January 5, 2017 /PRNewswire/ --
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 Authorisation 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 licence agreement in August 2013 to develop and commercialise
KRN23.
"Based on the positive results from multiple studies in
paediatric and adult patients with XLH, we are pursuing conditional
marketing authorisation 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
characterised by inadequate mineralisation 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 paediatric 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 characterised 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 programme studying KRN23 in adults and Phase 2 and
Phase 3 studies in paediatric patients with XLH are ongoing. KRN23
is also being developed for tumour-induced osteomalacia (TIO), a
disease characterised by typically benign tumours 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
licence 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 commercialisation 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 Kirin International PLC (KKI) is a subsidiary of Kyowa
Hakko Kirin and is a rapidly growing specialty pharmaceutical
company engaged in the development and commercialisation 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 centred 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 realise 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:
http://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
programmes, 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
programmes, 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
programmes, 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.