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Synageva BioPharma
, a biopharmaceutical
company developing therapeutic products for rare diseases, today announced
that the U.S. Food and Drug Administration (FDA) granted Breakthrough
Therapy designation to sebelipase alfa for the treatment of early onset
lysosomal acid lipase deficiency (LAL Deficiency), also known as Wolman
disease. The designation was based on data generated from clinical trials
with sebelipase alfa in patients with early onset LAL Deficiency. The FDA
also confirmed that late onset LAL Deficiency is "a serious and life
threatening disease or condition" and that Breakthrough Therapy designation
could be obtained for this aspect of the disease with additional clinical
information.
According to the FDA, Breakthrough Therapy designation is intended to
expedite the development and review of drugs for serious or life-threatening
conditions. The criteria for Breakthrough Therapy designation require
preliminary clinical evidence that demonstrates the drug may have
substantial improvement on at least one clinically significant endpoint over
available therapy. A Breakthrough Therapy designation conveys all of the
fast track program features, as well as more intensive FDA guidance on an
efficient drug development program.
"We are pleased that the FDA designated sebelipase alfa as Breakthrough
Therapy for patients with early onset LAL Deficiency, or Wolman disease, "
said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief
Medical Officer of Synageva. "We are deeply aware of the devastating impact
this disease has on infants who often die within the first six months of
life because of this disease. Our ongoing Phase 2/3 trial delivers hope for
these infants and their families. We continue to progress site activation
and patient enrollment in both this trial and the global Phase 3 ARISE trial
in children and adults, and look forward to working closely with the FDA to
support approval of the drug in an efficient manner."
About Synageva's lead programs sebelipase alfa for LAL Deficiency and
SBC-103 for MPS IIIB
LAL Deficiency is a rare autosomal recessive lysosomal storage disorder
(LSD) caused by a marked decrease in LAL enzyme activity. Late onset LAL
Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), is an
underappreciated cause of cirrhosis and accelerated atherosclerosis in
children and adults. These complications are due to the buildup of fatty
material in the liver and blood vessel walls as a result of decreased LAL
enzyme activity. Early onset LAL Deficiency, sometimes called Wolman
disease, is the most rapidly progressive form of LAL Deficiency and is
usually fatal within the first six months of life. Affected infants develop
severe malabsorption, growth failure and liver failure. There are no
approved therapies for LAL Deficiency.
Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme
under development by Synageva as an enzyme replacement therapy for LAL
Deficiency. Synageva is evaluating sebelipase alfa in global clinical trials
for both early and late onset LAL Deficiency. Sebelipase alfa has been
granted orphan designation by the FDA, the European Medicines Agency (EMA),
and the Japanese Ministry of Health, Labour and Welfare. Additionally,
sebelipase alfa received fast track designation by the FDA, and Breakthrough
Therapy designation by the FDA for early onset LAL Deficiency.
The mucopolysaccharidoses (MPS) consist of a group of rare LSDs caused by a
deficiency of enzymes needed to break down complex sugars called
glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo
syndromes) share complications with other MPS diseases but represent a
clinically distinct subset with marked central nervous system degeneration.
Mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome)
is caused by a marked decrease in alpha-N-acetyl-glucosaminidase (NAGLU)
enzyme activity which leads to the buildup of abnormal sugars called heparan
sulfate disaccharides (HSD) in the brain and other organs. The accumulation
of abnormal HSD, particularly in the central nervous system, leads to severe
cognitive decline, behavioral problems, speech loss, increasing loss of
mobility, and premature death. There are no approved therapies for MPS IIIB.
SBC-103 is a recombinant form of the human NAGLU enzyme under development by
Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing
approaches, SBC-103 reduced HSD substrate storage in the brains, liver and
kidney tissues in an MPS IIIB animal model. SBC-103 has been granted orphan
designation by the FDA and the EMA. Synageva plans to enter SBC-103 into
human clinical trials for MPS IIIB during the first half of 2014.
About Synageva BioPharma Corp.
Synageva is a biopharmaceutical company focused on the discovery,
development, and commercialization of therapeutic products for patients with
life-threatening rare diseases and unmet medical need. Synageva has several
protein therapeutics in its drug development pipeline.
Synageva routinely posts information that may be important to investors in
the "Investor Relations" section of our web site at www.synageva.com.
Synageva encourages investors and potential investors to consult our web
site regularly for important information about us.
Forward-Looking Statements
This news release contains "forward-looking statements". Such statements
generally can be identified by the use of words such as "anticipate,"
"expect," "plan," "could," "intend," "believe," "may," "will," "estimate, "
"forecast," "project," or words of similar meaning. These forward-looking
statements address, among other matters, our plans for seeking regulatory
approval for sebelipase alfa, our plans to work with the FDA to support
regulatory approval in an efficient manner, obtaining the Breakthrough
Therapy designation for the late onset form of LAL Deficiency, our hope that
we may be able to assist the infants and our plans to enter into human
clinical trials for MPS IIIB. Many factors may cause actual results to
differ materially from forward-looking statements, including inaccurate
assumptions and a broad variety of risks and uncertainties, some of which
are known, including an accurate understanding of the implications of
Breakthrough Therapy designation which cannot be determined at this time,
our ability to rely on or utilize this Breakthrough Therapy designation in a
meaningful way with respect to regulatory approval of sebelipase alfa, the
generation of the additional clinical information necessary for Breakthrough
Therapy designation in the late onset form of LAL Deficiency and the risks
associated with transitioning our preclinical MPS IIIB program to human
clinical trials and those additional risks identified under the heading
"Risk Factors" in the Company's Annual Report on Form 10-Q filed with the
Securities and Exchange Commission (the "SEC") on May 7, 2013, and other
filings Synageva periodically makes with the SEC, and others of which are
not known. No forward-looking statement is a guarantee of future results or
events, and investors should avoid placing undue reliance on such
statements. Synageva undertakes no obligation to update any forward-looking
statements, whether as a result of new information, future events or
otherwise. Our business is subject to substantial risks and uncertainties,
including those referenced above. Investors, potential investors, and others
should give careful consideration to these risks and uncertainties.
"Dedicated to Rare Diseases(R)" is a registered trademark. "Synageva
BioPharma(TM)" is a trademark of Synageva BioPharma Corp.
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