NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing orphan therapeutics for rare gastrointestinal and endocrine disorders, is joining the National Organization for Rare Disorders (NORD) and other organizations around the world today to observe World Rare Disease Day on February 29, 2012. This observance spotlights the nearly 30 million people in the United States and millions more worldwide who are living with a rare disorder, many of whom have difficulty getting the right care because of a lack of education about these conditions.
“Rare Disease Day reminds us that there are millions of people who are waiting for treatments for their rare disorders,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “As a company dedicated to developing treatments for orphan gastrointestinal and endocrine disorders, NPS salutes the researchers, patients, advocates, and healthcare providers who are working tirelessly to make a meaningful difference in the lives of those affected by rare disorders.”
In the U.S., any disease affecting fewer than 200,000 Americans is considered rare. According to the National Institutes of Health (NIH), there are approximately 7,000 such diseases affecting nearly 30 million Americans. While nearly one in 10 Americans lives with a rare disease, only about 200 of the diseases classified as rare have FDA-approved treatments.
“Everyone knows someone with a rare disease but many don’t appreciate the support these patients need," said Peter L. Saltonstall, president and CEO of NORD. "We have been celebrating Rare Disease Day for five years, and in that time it has become a truly global movement. With the help of partners such as NPS Pharmaceuticals, we can empower people on nearly every continent to ensure that patients living with rare diseases are heard.”
About Rare Disease Day
Launched in Europe five years ago, World Rare Disease Day was recognized last year in 60 countries, each of which has a national sponsor. NORD is the sponsor in the U.S.
On Rare Disease Day 2012, activities in the U.S. will include a “Handprints Across America” campaign to create a gallery of photos on the Rare Disease Day website; educational materials for classroom teachers; and a nationwide blitz of patient photos, stories and videos to increase awareness of specific rare diseases and the challenges of living with a rare disease. In addition, several special events are planned, including a scientific symposium at the National Institutes of Health (NIH) and a Rare Disease Patient Advocacy Day in conjunction with the Food and Drug Administration (FDA).
In the U.S., the coalition supporting Rare Disease Day includes patient organizations and advocacy groups, medical professionals and associations, government agencies, researchers, and companies developing treatments for rare diseases. More than 500 patient organizations, government entities, research institutions, and companies developing treatments have signed up as Rare Disease Day Partners on the national website hosted by NORD (www.rarediseaseday.us).
About Short Bowel Syndrome
Short bowel syndrome, or SBS, is a highly disabling condition that can lead to serious life-threatening complications and impair a patient's quality-of-life. SBS typically arises after extensive resection of the bowel due to Crohn's disease, ischemia or other conditions. SBS is associated with intestinal failure and the inability to absorb sufficient nutrients and/or fluids through the gastrointestinal tract. Patients with SBS are highly prone to malnutrition, diarrhea, dehydration, and an inability to maintain weight due to the reduced intestinal capacity to absorb macronutrients, water, and electrolytes. As a result, many patients require the chronic use of parenteral nutrition (PN) and intravenous (IV) fluids to supplement their nutritional needs and stabilize their hydration. PN/IV fluids bypass the digestive tract and are delivered directly into the bloodstream through a central venous catheter. Although PN/IV can meet basic nutrition and fluid requirements, it does not improve the body's ability to absorb nutrients.
The long-term use of PN/IV fluids is associated with shortened life span and life-threatening complications such as blood clots and liver damage. The development of PN-associated liver disease predisposes patients to an increased incidence of sepsis, increased mortality rates, and the potential to develop irreversible liver damage. Patients on parenteral support often experience a poor quality of life with difficulty sleeping, frequent urination, and loss of independence.
There are an estimated 10,000 to 15,000 SBS patients in the U.S. who are dependent on parenteral nutrition and intravenous fluids.
Hypoparathyroidism is a rare disorder in which the body produces insufficient levels of parathyroid hormone, the principal regulator of calcium and phosphorus. When the body has too little parathyroid hormone, blood calcium levels drop and phosphorus levels increase, which can cause muscular and neurological symptoms, as well as bone impairments. There is no approved hormone replacement for hypoparathyroidism. It is one of the few remaining hormone deficiency syndromes in which replacement therapy using the native hormone is not clinically available. Hypoparathyroidism is currently managed with large doses of calcium supplementation and active vitamin D therapy to raise the calcium levels in the blood and reduce the severity of symptoms. Over time, calcium may build up in the body and result in serious health risks, including calcifications in the kidneys, heart or brain.
About NPS Pharmaceuticals
NPS Pharmaceuticals is a biopharmaceutical company focused on bringing orphan products to patients with rare disorders and few, if any, therapeutic options. NPS is advancing two late-stage registration programs. A New Drug Application is undergoing FDA review for Gattex® (teduglutide) as a treatment for adult short bowel syndrome (SBS) and a Phase 3 registration study has been completed for Natpara™ (recombinant human parathyroid hormone (rhPTH [1-84]) in adult hypoparathyroidism. NPS’ earlier stage pipeline includes two calcilytic compounds, NPSP790 and NPSP795, with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia with hypercalciuria (ADHH). NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Kyowa Hakko Kirin, Nycomed, and Janssen Pharmaceuticals, Inc.
“NPS”, “NPS Pharmaceuticals”, and “GATTEX” are the company’s registered trademarks. All other trademarks, trade names or service marks appearing in this press release are the property of their respective owners.
Statements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements are based on the company's current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Risks associated to the company’s business include, but are not limited to, the risks associated with any failure by the company to successfully complete its preclinical and clinical studies within the projected time frames or not at all, the risk of not gaining marketing approvals for GATTEX and NPSP558, the risks associated with the company’s strategy, as well as other risk factors described in the company’s periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Form 10-Qs. All information in this press release is as of the date of this release and NPS undertakes no duty to update this information.