Synageva's Sebelipase Alfa Receives Breakthrough Therapy Designation for Early Onset LAL Deficiency from the U.S. Food and Drug Administration
LEXINGTON, Mass.--(BUSINESS WIRE)--
BioPharma Corp. (Synageva) (NASDAQ:GEVA), 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
About Synageva's lead programs sebelipase alfa for LAL Deficiency and
SBC-103 for MPS IIIB
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.
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
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.
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.
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®" is a registered trademark. "Synageva
BioPharma™" is a trademark of Synageva BioPharma Corp.
Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947
Source: Synageva BioPharma Corp.
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