TOKYO, LONDON, and NOVATO,
California, December 15, 2017
/PRNewswire/ --
If approved,
burosumab would be the first targeted
disease-modifying treatment for XLH
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
authorisation 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 authorisation. 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 authorisation
requires fulfilment of specific obligations related to the
completion of ongoing clinical studies of burosumab in paediatric
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 commercialisation of burosumab
globally, based on the collaboration and licence agreement between
Kyowa Hakko Kirin and Ultragenyx.
Burosumab Regulatory Status
The EMA's scientific committee adopted a Positive Opinion
recommending the conditional marketing authorisation 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 tumour-induced osteomalacia (TIO), diseases characterised 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 paediatric and
adult patients with XLH, and has set a Prescription Drug User Fee
Act (PDUFA) action date of April 17,
2018.
A clinical programme studying burosumab in adults and paediatric
patients with XLH is ongoing. Burosumab is also being developed for
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.
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 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.
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 commercialisation 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
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.
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.
SOURCE Kyowa Kirin International PLC