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Synageva BioPharma Joins NORD, EURORDIS, And The Global Genes Project In Support Of Rare Disease Day 2015

LEXINGTON, Mass., Feb. 27, 2015 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a 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 "Living with a Rare Disease" to help convey the challenges shared by the families and friends whose daily lives are impacted and who are living day-by-day, hand-in-hand with rare disease patients.

"One in 10 Americans has a rare disease and Rare Disease Day is an important day to raise awareness that helps to improve the lives of these 30 million Americans," said Peter L. Saltonstall, President and CEO of NORD, a leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases, and sponsor of Rare Disease Day in the U.S.

Each year, Synageva joins efforts with The Global Genes Project, a leading non-profit organization for rare and genetic diseases, in a volunteer fundraiser with proceeds to benefit the Support Organization for Lysosomal Acid Lipase Deficiency Advocacy, Care and Expertise (LAL Solace) organization and other LAL Deficiency patient organizations and patient advocacy groups.

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 globally, go to

For more information about NORD, go to

For more information about EURORDIS, go to

For more information about The Global Genes Project, go to

For more information about LAL Solace, go to

Kanuma™ (sebelipase alfa) and LAL Deficiency

LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test.  LAL Deficiency causes progressive and multisystemic organ damage including cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications.  LAL Deficiency often manifests in childhood but can be diagnosed at all ages.  LAL Deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.

Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency.  Kanuma has been granted orphan designation by the FDA, the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare.  Additionally, Kanuma received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.  The FDA accepted for review the BLA for Kanuma and granted the company's request for Priority Review.  The EMA validated the Marketing Authorization Application (MAA) for Kanuma and granted the company's request for accelerated assessment.

SBC-103 and MPS IIIB

Mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is a rare disease caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfate (HS) in the brain and other organs.  The accumulation of abnormal HS in multiple cells and tissues leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.  SBC-103 is a recombinant form of natural human NAGLU designed to replace the missing (or deficient) NAGLU enzyme and dosing with SBC-103 in patients with MPS IIIB recently began as part of a Phase 1/2 study.

SBC-103 has favorable properties for enabling cellular uptake and has shown the ability to overcome the challenges previously encountered in producing recombinant human NAGLU.  The advancement of SBC-103 towards the clinic was supported by preclinical studies demonstrating that intravenously administered SBC-103 was able to cross the blood-brain barrier and reduce HS storage in the brain in an MPS IIIB animal model.  In addition, SBC-103 demonstrated transport across an in vitro model of the blood-brain barrier and distributed into the CSF in non-human primate studies.

The company is conducting natural history studies in MPS IIIB.  These include a retrospective natural history study of deceased MPS IIIB patients that began in July 2013 and a prospective, longitudinal natural history study in living MPS IIIB patients that began in September 2014.  Natural history studies can help build an understanding of the manifestations and progression of disease and provide insights into biomarkers and other measures that may be useful as clinical outcomes.

SBC-103 was granted orphan designation by the FDA in April 2013 and the European Medicines Agency (EMA) in June 2013 and received Fast Track designation by the FDA in January 2015.

About Synageva

Synageva is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with rare diseases. The company's pipeline consists of protein therapeutic programs for rare diseases with unmet medical need which are currently at various stages of development.  The company is planning for a global launch of Kanuma for the treatment of LAL Deficiency and initiated a Phase 1/2 trial with its second, first-mover program, SBC-103 for patients with MPS IIIB.  The company's third, first-mover program, SBC-105, is an enzyme replacement therapy in preclinical development for rare disorders of calcification, including generalized calcification in infants (GACI).  In addition to these first-mover programs, the pipeline also consists of opportunities that leverage the company's manufacturing platform and other capabilities to create potentially bio-superior treatments for patient populations where there is still unmet medical need.  The company has recently produced enzymes targeting Hunter syndrome, Fabry disease and Pompe disease with expression levels and activity that support further preclinical development.

Synageva routinely posts information that may be important to investors in the Investor Relations section of the company's website at  Synageva encourages investors and potential investors to consult this website regularly for important information about the company.

Medical information regarding Kanuma and LAL Deficiency is available by email at

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, the planned launch of Kanuma.  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, such as,  the risk that the outcomes of our clinical trials may not support registration or further development of our product candidates due to safety, efficacy or other reasons, the content and timing of decisions by the FDA and other regulatory authorities, and the risks identified under the heading "Risk Factors" in the Company's annual report on Form 10-K filed with the Securities and Exchange Commission (SEC) on February 26, 2015 and other filings Synageva periodically makes with the SEC, and others of which are not known.  Preclinical and clinical trial data are subject to differing interpretations, and regulatory agencies, as well as medical and scientific experts, may not share Synageva's views regarding these data or its implications.  Synageva may encounter problems or delays in preclinical and clinical development and the regulatory process.  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.

"Synageva BioPharma™" and "Kanuma™" are trademarks, and "Dedicated to Rare Diseases®" is a registered trademark, of Synageva BioPharma Corp. "National Organization for Rare Disorders™" and "NORD™" are trademarks of NORD.  "European Organisation for Rare Diseases™" and EURORDIS™" are trademarks of EURORDIS. "The Global Genes Project™" is a trademark of Global Genes.


SOURCE Synageva BioPharma Corp.
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