Synageva BioPharma™ Joins NORD, EURORDIS, and The Global Genes Project in
Support of Rare Disease Day 2013
LEXINGTON, Mass. -- February 28, 2013
Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a clinical stage
biopharmaceutical company developing therapeutic products for rare diseases,
joins the National Organization for Rare Disorders (NORD), the European
Organisation for Rare Diseases (EURORDIS), The Global Genes Project, and
others around the world in observing Rare Disease Day.
On the last day of February each year, patient organizations, industry, and
other participants conduct special events to raise awareness and advance
policies to improve conditions for people living with rare diseases. The
theme, or slogan, this year for Rare Disease Day is “Rare Disorders Without
Borders” to help convey the global solidarity of the rare disease community
which shares many of the same medical, social and economic challenges. In the
U.S., this year’s Rare Disease Day also coincides with the 30th anniversary of
the Orphan Drug Act, which provides incentives to encourage companies to
develop treatments for rare diseases, and of the founding of NORD, which was
established by patient advocates in 1983.
“There are nearly 30 million Americans—and millions more around the
world—affected by rare diseases,” said Peter L. Saltonstall, President and CEO
of NORD. “Everyone knows someone with a rare disease. But, while many of these
diseases are serious and lifelong, most have no treatment and many are not
even being studied by researchers.”
“More than half of the people who have rare diseases are children,”
Saltonstall said. “Challenges faced by patients and their families include
delayed diagnosis, few treatment options, and difficulty finding medical
experts. Many rare diseases have no approved treatment.”
Synageva is proud to join efforts during this year’s Rare Disease Day with The
Global Genes Project, a leading non-profit organization for rare and genetic
diseases. Along with displaying the signature Blue Denim Genes Ribbon™
associated with The Global Genes Project, Synageva employees will participate
in a volunteer fund-raiser with proceeds to benefit the Support Organization
for Lysosomal Acid Lipase Deficiency Advocacy, Care and Expertise (LAL
Solace), a patient advocacy group for individuals affected by lysosomal acid
lipase deficiency (LAL Deficiency).
“We are profoundly aware of the challenges facing patients and their families
who suffer from rare diseases,” said Sanj K. Patel, President and CEO of
Synageva. “We are deeply driven and motivated to help these often overlooked
and underserved individuals and are focused on making a meaningful impact on
their lives. We applaud the efforts of NORD, EURORDIS, The Global Genes
Project, LAL Solace and others to help raise awareness of rare diseases around
In the U.S., a rare disease is one that affects fewer than 200,000 Americans,
and in Europe, less than one in 2,000 citizens. According to EURORDIS,
approximately 80% of rare diseases have a genetic origin. Approximately 7,000
rare diseases have been identified to date, affecting nearly 30 million
Americans and 30 million Europeans.
For more information about Rare Disease Day activities in the U.S., go to
For information about Rare Disease Day activities globally, go to
For more information about The Global Genes Project™, go to
For more information about LAL Solace, go to www.lalsolace.org.
About Synageva’s Lead Program
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, a
lysosomal storage disorder (LSD). Synageva is currently evaluating sebelipase
alfa in global clinical trials for both early and late onset LAL Deficiency.
Sebelipase alfa has been granted orphan designations by the U.S. Food and Drug
Administration (FDA), the European Medicines Agency, and the Japanese Ministry
of Health, Labour and Welfare. Additionally, sebelipase alfa received “fast
track” designation by the FDA.
About LAL Deficiency
LAL Deficiency is a rare autosomal recessive LSD caused by a marked decrease
in LAL enzyme activity. Late onset LAL Deficiency, sometimes called
Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In
these patients, the buildup of fatty material in the liver and blood vessel
walls may lead to liver cirrhosis, liver failure and accelerated
atherosclerotic events. Early onset LAL Deficiency, sometimes called Wolman
disease, affects infants and is characterized by severe malabsorption, growth
failure and liver failure, and is usually fatal within the first six months of
life. There are no approved pharmacological therapies for LAL Deficiency.
Success with stem cell and liver transplant appears to be limited by
procedure-related morbidity and mortality.
About Synageva BioPharma Corp.
Synageva is a clinical stage 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. The company
has a team with a proven record of bringing therapies to patients with rare
Further information regarding Synageva BioPharma Corp. is available at
This news release and oral statements made from time to time by Synageva
representatives in respect of the same subject matter may contain
“forward-looking statements” under the provisions of the Private Securities
Litigation Reform Act of 1995. Such statements can be identified by
introductory words such as “expects,” “plans,” “intends,” “believes,” “will,”
“estimates,” “forecasts,” “projects,” or words of similar meaning and by the
fact that they do not relate strictly to historical or current facts. 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 those identified under the
heading “Risk Factors” in the Company’s prospectus supplement filed with the
Securities and Exchange Commission (the “SEC”) on January3, 2013, and other
filings Synageva periodically makes with the SEC and others of which are not.
“Dedicated to Rare Diseases®” is a registered trademark and “Synageva
BioPharma™” is a trademark of Synageva BioPharma Corp.
Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947
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