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Sigma-Tau Pharmaceuticals Stands United with Patient Organizations on Rare Disease Day.

GAITHERSBURG, Md. -- Sigma-Tau Pharmaceuticals, Inc., a part of the Sigma Tau Group Rare Disease Franchise, today announced its support of Rare Disease Day, a global campaign to raise awareness of rare diseases and their impact on patients and families. Rare Disease Day is an opportunity for patient organizations, researchers, industry and governments worldwide to improve the understanding, diagnosis and treatment of rare diseases. The annual celebration is organized by the European Organization for Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD).

This year also marks the 30th anniversary of the passage of the Orphan Drug Act. This landmark legislation encouraged the discovery and development of innovative therapies for rare disorders in the United States and led to the adoption of similar policies worldwide.

"Sigma-Tau was one of the first companies to receive an orphan drug designation, and for nearly 30 years has remained steadfast in its mission of creating novel medicines for patients with rare diseases.", declared Dave Lemus, Chief Operating Officer of Sigma-Tau Pharmaceuticals, Inc. "On this special day, Sigma-Tau reaffirms its commitment to developing and delivering innovative therapies for patients with rare diseases and urge continued collaboration among patient organizations, researchers, governments and industry to meet the needs of these communities."

In October 2012, Sigma-Tau received U.S. Food and Drug Administration (FDA) approval for Cystaran[TM] (cysteamine ophthalmic solution) 0.44%, a novel treatment for corneal cystine crystal accumulation as a result of cystinosis, a rare, genetic lysosomal-storage disease. Sigma-Tau developed Cystaran in partnership with the National Eye Institute, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Human Genome Research Institute, and in cooperation with three cystinosis patient advocacy groups: the Cystinosis Research Network, the Cystinosis Foundation and the Cystinosis Research Foundation. The Cystaran project was supported in part by an orphan drug grant from the FDA Office of Orphan Products Development.

"Collaborative research can produce urgently needed therapies for rare diseases. Researchers from three different institutes within NIH partnered with Sigma-Tau to develop Cystaran, with support from the FDA and input from patient groups," said William A. Gahl, M.D., Ph.D., Clinical Director, National Human Genome Research Institute, NIH. "On Rare Disease Day, we remind patients and families affected by rare diseases that they are not alone."

About Rare Diseases

In the United States, a disease is defined as rare if it affects fewer than 200,000 people. While individually each of these approximately 7,000 diseases is rare, together they impact nearly 30 million Americans, or one in 10 people, according to the NIH. Some of the challenges of living with a rare disease may include: difficulty getting a timely, accurate diagnosis; too little research; too few treatments; reimbursement or other issues affecting access to treatments; difficulty finding medical experts, and a sense of isolation.

About Rare Disease Day

Rare Disease Day was launched by EURORDIS in 2008. Today, it is observed in more than 60 countries, with a national sponsor in each country. NORD is the sponsor in the United States. Each year, a global planning team selects a theme for Rare Disease Day. The theme for 2013 is "Rare Disorders without Borders." More information is available at

About Cystaran[TM]

The clinical safety and efficacy of Cystaran[TM] was previously evaluated in controlled clinical trials conducted by the NIH, in approximately 300 patients. Results of these studies support the use of ophthalmic cysteamine as an effective treatment of corneal cystine crystals. The most frequently reported ocular adverse reactions, occurring in 10% of patients, were sensitivity to light, redness, eye pain/irritation, headache and visual field defects. Please click here for full prescribing information for Cystaran[TM].

About Sigma-Tau Pharmaceuticals, Inc.

Sigma-Tau Pharmaceuticals, Inc. is a U.S. based, wholly owned subsidiary of the sigma-tau Group, and is dedicated to the global development and commercialization of medicines for patients with rare diseases. Sigma-Tau Pharmaceuticals, Inc. is based in Gaithersburg, Maryland. Since 1989, the company's products have been focused on kidney disease, certain genetic disorders and cancers. With more than 7,000 identified rare diseases that affect approximately 30 million patients in the U.S. alone, Sigma-Tau places its considerable scientific resources behind the development and commercialization of compounds that benefit the few. The company has a substantial development program focused on transplant, cancer, inherited genetic disorders, malaria, and other areas of unmet medical need. For more information about the company, visit
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Publication:Business Wire
Geographic Code:1U5MD
Date:Feb 28, 2013
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