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NPS Pharmaceuticals Recognized by National Organization for Rare Disorders for Gattex® as an Innovative Treatment for Patients with Short Bowel Syndrome

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Thursday, May 16, 2013

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NPS Pharmaceuticals, Inc., a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, announced that the company was honored by the National Organization for Rare Disorders (NORD) for the FDA approval of Gattex® (teduglutide [rDNA origin]) for injection, which is indicated for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support. SBS is a rare and potentially fatal gastrointestinal disorder in which patients are unable to absorb enough nutrients and fluids through the intestine from the foods they eat.

“We are honored to be recognized by NORD and proud to have contributed to making a significant difference in the lives of patients with Short Bowel Syndrome” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “The mission of NORD and dedication of their leadership has proven to be an essential catalyst in improving the lives of patients with rare diseases over the past three decades.”

The event commemorated the 30th anniversary of the enactment of the Orphan Drug Act and the formation of NORD, while honoring companies and individuals who made significant contributions to the rare disease community in 2012. NPS was honored for bringing Gattex, the first major advance for the long-term treatment of SBS in nearly 40 years, to adult patients with the rare disorder who are dependent on parenteral support. A combination of a nutritional supplement and essential fluid, parenteral support is administered intravenously, for up to seven days per week. The long-term use of parenteral support can be associated with serious and sometimes life-threatening complications. Gattex may help the remaining bowel absorb more and consequently reduce the need for parenteral support.

“We at NORD would like to congratulate NPS for its innovative and important work on behalf of short bowel syndrome patients in the U.S. and for making a particularly significant contribution by bringing a new treatment option to these patients,” said Peter L. Saltonstall, president and chief executive officer of NORD.

The event was held at the Mellon Auditorium in Washington, DC on Tuesday, May 14, 2013 to honor leadership in public policy and pioneers in the patient community, government and industry for years of service to rare disease patients and their families. Honorees in Public Policy will include William Corr, Deputy Secretary, U.S. Department of Health and Human Services; The Honorable Nancy Kassebaum, U.S. Senate, retired; and The Honorable Henry Waxman, U.S. House of Representatives, retired. Those to be honored For Vision and Pioneering Guidance include Stephen C. Groft, PharmD; Marlene Haffner, MD, MPH; Lars Uno Larsson; Abbey S. Meyers; and Jess G. Thone, MD.

About Short Bowel Syndrome

Short bowel syndrome (SBS) is a highly disabling condition that can impair a patient's quality of life and lead to serious life-threatening complications. SBS typically arises after extensive resection of the bowel due to Crohn's disease, ischemia or other conditions. SBS patients often suffer from malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight loss due to the reduced intestinal capacity to absorb nutrients, water and electrolytes. The usual treatment for SBS is nutritional support, including parenteral nutrition (PN) and/or intravenous (IV) fluids to supplement and stabilize nutritional needs.

Although PN can provide nutritional support for SBS patients, it does not improve the body's own ability to absorb nutrients. PN is associated with serious complications, such as infections, blood clots or liver damage, and the risks increase the longer patients are on PN. Patients on PN often experience poor quality of life with difficulty sleeping, and frequent urination, and patients receiving chronic PN often experience a loss of independence.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide. The company’s lead product, Gattex® (U.S.)/Revestive® (EU) (teduglutide [rDNA origin]) for injection is approved for adult Short Bowel Syndrome (SBS) patients who are dependent on parenteral support. NPS is also developing Natpara® (rhPTH [1-84]) for the treatment of adult hypoparathyroidism and, subject to the resolution of certain manufacturing issues, expects to submit its Biologic License Application (BLA) to the FDA in 2013.

NPS's earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia (ADH). NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin.

“NPS,” “NPS Pharmaceuticals,” “Gattex,” “Natpara,” “Revestive,” “Preotact,” and “NPS Advantage” are the company's trademarks. All other trademarks, trade names or service marks appearing in this press release are the property of their respective owners.

Susan M. Mesco
Source: NPS Pharmaceuticals, Inc.
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