BRISBANE, Calif., Feb. 27, 2015 (GLOBE NEWSWIRE) -- Hyperion Therapeutics, Inc. (Nasdaq:HPTX) today joins with patients and their families, caregivers, researchers, the biopharmaceutical industry and others in recognizing and supporting Rare Disease Day 2015. Observed annually on the last day of February, Rare Disease Day is observed in more than 80 nations and is led internationally by the European Organization of Rare Diseases (EURORDIS) and the National Organization for Rare Disorders (NORD) in the U.S. Hyperion is proud to support this effort to improve awareness and education of rare diseases.
"At Hyperion, we are dedicated to championing a future for patients living with rare, or orphan, diseases," said Donald J. Santel, president and chief executive officer of Hyperion Therapeutics, Inc. "There are millions of patients worldwide challenged with a rare disease and we are honored to support EURORDIS and NORD in their mission to elevate understanding of rare diseases, and to call attention to the challenges faced by these patients and their communities."
According to the U.S. National Institutes of Health, a disease is rare if it affects fewer than 200,000 people. Nearly 1 in 10 Americans live with a rare disease—affecting 30 million people—and two-thirds of these patients are children. There are nearly 7,000 identified rare diseases, and with only approximately 450 U.S. Food and Drug Administration medical treatments approved for all of these diseases, many of these patients may receive no treatment for their disorders.
Hyperion Recognizes Rare Disease Day with Low Protein Potluck
Hyperion currently markets two FDA approved therapies for the chronic treatment of urea cycle disorders, or UCDs. UCDs are a collection of inherited metabolic disorders in which affected patients suffer from high levels of systemic ammonia, a potent neurotoxin.
Ammonia is produced via normal protein digestion; the body normally detoxifies it by converting it through a series of enzymatic steps to urea, which is excreted in urine. Throughout their lives, UCD patients are at risk of recurrent hyperammonemic crises in which ammonia levels rise, and patients can develop complications ranging from nausea, vomiting and headache to coma and death. As part of their treatment regimen, most patients with UCD need to maintain a low protein diet.
In support of patients suffering from UCDs and in observation of Rare Disease Day 2015, Hyperion employees organized a low protein potluck at the company's headquarters. Through this event, Hyperion employees experienced for one afternoon a part of the daily lives of patients living with UCD and gained an appreciation for the thought and extra effort UCD patients need to put into the preparation of their daily meals. To watch Hyperion employee testimonials on the low-protein potluck please visit: https://www.ucdsupport.com/ by March 13, 2015.
On February 1, 2013, the FDA approved Hyperion's RAVICTI (glycerol phenylbutyrate) Oral Liquid for use as a nitrogen-binding agent for chronic management of adult and pediatric patients ≥ 2 years of age with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. The company also acquired BUPHENYL® (sodium phenylbutyrate) Tablets and Powder for the treatment of UCDs.
A disease or disorder is defined as rare in the U.S. when it affects fewer than 200,000 Americans at any given time. There are nearly 7,000 such diseases affecting nearly 30 million Americans, according to the National Institute of Health (NIH).
About Hyperion Therapeutics
Hyperion Therapeutics, Inc. is a commercial-stage biopharmaceutical company committed to developing and delivering life-changing treatments for orphan diseases. The company's first commercial product, RAVICTI (glycerol phenylbutyrate) Oral Liquid, was approved in February 2013 and is currently being marketed in the United States. The company also owns worldwide rights to BUPHENYL (sodium phenylbutyrate) Tablets and Powder, which it markets in the U.S. The compound is also marketed internationally through business partners. In addition, the company is developing glycerol phenylbutyrate for the potential treatment of hepatic encephalopathy. For more information, please visit www.hyperiontx.com.