LEXINGTON, Mass. -- (BUSINESS WIRE) -- 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 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.
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.