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Sigma-Tau Receives Innovation Award From The National Organization for Rare Disorders for Its Advancements in Rare Diseases

GAITHERSBURG, Md., May 15, 2013 /PRNewswire/ -- Sigma-Tau Pharmaceuticals, Inc. announced today that the Company was recognized last evening as a rare disease pioneer at the 30th anniversary celebration of The National Organization for Rare Disorders (NORD).  During the event, Sigma-Tau received an innovation award for the development of CYSTARAN™ (cysteamine ophthalmic solution) 0.44% --  the first and only approved therapy for the treatment of corneal cystine crystal accumulation in patients with cystinosis.

"NORD applauds Sigma-Tau Pharmaceuticals, NIH researchers, and patient organizations for collaborating to develop CYSTARAN for the treatment of this debilitating eye complication of cystinosis," said Peter L. Saltonstall , President and Chief Executive Officer of NORD. "As one of the first companies to receive an orphan-drug designation nearly 30 years ago, Sigma-Tau is once again demonstrating its strong and enduring commitment to serving rare disease communities with innovative products and effective patient programs."

Sigma-Tau developed CYSTARAN in partnership with the National Institutes of Health (NIH) and in cooperation with the Cystinosis Foundation, the Cystinosis Research Foundation, and the Cystinosis Research Network. The U.S. Food and Drug Administration (FDA) approved CYSTARAN for the treatment of this painful and debilitating eye condition in October 2012.

"We applaud NORD and all award recipients for their dedication to bringing innovative therapies and new hope to patients with rare diseases," said Dave Lemus , Chief Executive Officer of Sigma-Tau. "We are privileged to receive this award and remain committed to serving the rare disease community by developing and delivering innovative therapies."

Patient assistance programs for CYSTARAN are available, including co-pay assistance for eligible patients and access to CYSTARAN therapy for uninsured or under-insured patients.  Patients, caregivers and physicians in the United States and Puerto Rico can access the CYSTARAN Hotline at 1-800-440-0473, or by visiting the Accredo website,

For full prescribing information for CYSTARAN, see

Safety: 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.

About Cystinosis
Cystinosis, which affects approximately 2,000 individuals worldwide, is a rare metabolic disease in which the amino acid cystine enters cells, but has no transporter out of the cells. The defect in transportation leads to formation of crystals within the cells, causing early cell death. Cystinosis slowly destroys numerous vital organs including the kidneys, liver, eyes, muscles and the brain.  Corneal cystine accumulation can lead to ocular complications such as squinting, foreign body sensations, changes in visual acuity, corneal haziness and photophobia (i.e., sensitivity to light). Other complications of cystinosis include muscle weakness, diabetes, hypothyroidism, difficulty swallowing and rickets.  

About Sigma-Tau Pharmaceuticals
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

SOURCE Sigma-Tau Pharmaceuticals, Inc.
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