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Hyperion Therapeutics Receives Orphan Drug Designation for HPN-100 for the Treatment of Urea Cycle Disorders
Date:5/5/2009

affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance, waiver of Prescription Drug User Fee Act (PDUFA) filing fees, and a seven-year period of U.S. marketing exclusivity if the drug is the first of its type approved for the specified indication or if it demonstrates superior safety, efficacy, or a major contribution to patient care versus another drug of its type previously granted the designation for the same indication.

About Urea Cycle Disorders

Urea cycle disorders are inherited, inborn errors of metabolism present in an estimated 1 in 10,000 births in the United States. Patients with urea cycle disorders are deficient in one of the key enzymes that comprise the urea cycle, the body's primary vehicle for removing ammonia, a potent neurotoxin, from the bloodstream. Onset may occur at any age depending on the severity of the disorder. Left untreated, urea cycle disorders can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.

About NUCDF

The National Urea Cycle Disorders Foundation is a nationally-recognized nonprofit organization dedicated to saving children and adults from the catastrophic effects of urea cycle disorders. The Foundation is a lifeline to families and medical professionals all over the world desperately seeking crisis intervention and treatment information. The Foundation's mission is to provide information, guidance and support to patients and their families, to educate healthcare professionals on the identification, diagnosis and treatment of UCDs, to raise awareness so that no child or adult ever goes undiagnosed, and to stimulate and support critical research activities and a CURE. The Foundation is headquartered in Pasadena, California. For mo
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SOURCE Hyperion Therapeutics, Inc.
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