“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 the world.”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 www.rarediseaseday.us. For information about Rare Disease Day activities globally, go to www.rarediseaseday.org. For more information about The Global Genes Project™, go to www.globalgenes.org. 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 diseases.
Further information regarding Synageva BioPharma Corp. is available at www.synageva.com.Forward-Looking Statements 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 January 3, 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.