NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, is joining the National Organization for Rare Disorders (NORD) and other organizations around the world today to observe World Rare Disease Day. In the U.S., the observance is led by NORD and focuses on the 25-30 million people living with a rare disease, many of whom have difficulty getting the right care because of a lack of education about these disorders.
This year also marks the 30th anniversaries of NORD and the Orphan Drug Act. The legislation, which was driven by a determined group of individuals that also founded NORD, encourages the development and commercialization of treatments for rare diseases, or “orphan” drugs. The act was designed to provide financial incentives, including seven-year market exclusivity for an FDA-approved treatment and an accelerated review process for drug candidates that qualify. Shortly after the act was signed, NORD was officially formed and 30 years later, the organization is widely recognized as the leading advocate for awareness and recognition of the challenges faced by people living with rare diseases.
“Considering about one in every ten people in the U.S. is living with some form of rare disease and as many as 60 million worldwide, Rare Disease Day is an observance that truly transcends cultures and borders and is deserving of international attention,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “We at NPS recognize NORD as a noble champion of patient advocacy in the rare disease community and applaud the organization for its 30 years of service.”
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, yet less than 5 percent of these disorders have FDA-approved therapies.
“While we have made great strides in the last thirty years to raise international awareness and recognition of rare diseases, we have a renewed commitment toward our goal of identifying, treating, and ultimately curing rare disorders through programs encouraging education, advocacy, research and service,” said Peter L. Saltonstall, president and CEO of NORD. “As we celebrate this milestone anniversary, NORD is proud to have the support of organizations such as NPS Pharmaceuticals who are bringing rare disease treatments to patients in need.”
About Rare Disease Day
Launched in Europe in 2008, World Rare Disease Day was recognized last year in more than 60 countries, each of which has a national sponsor. NORD is the sponsor in the U.S.
Each year, Rare Disease Day organizers around the world agree beforehand on a slogan to express the specific message for that year. For 2013, the slogan is “Rare Disorders Without Borders” to convey the global solidarity of the rare disease community. Rare disease patients all over the world face many of the same medical, social and economic challenges. Together, in solidarity, the global rare disease community is determined to build a better world for patients. Visit the Rare Disease Day website (http://www.rarediseaseday.org/article/rare-disorders-solidarity-has-no-border) to learn more.
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).
The National Organization for Rare Disorders (NORD) represents the nearly 30 million Americans affected by rare diseases. Founded in 1983, NORD is a non-profit organization providing programs of education, advocacy, research, and patient services. NORD administers patient assistance programs to help uninsured or under-insured individuals obtain medications they could not otherwise afford. It also provides information to patients and their families, research grants and fellowships, and advocacy on important public policy issues. Follow NORD at www.rarediseases.org and on Twitter at @RareDiseases.
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.
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 pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide. The company’s lead product, Gattex® 0.05 mg/kg/d (teduglutide [rDNA origin]) for injection is FDA-approved for the treatment of adult patients with short bowel syndrome (SBS) who are dependent on parenteral support. NPS is also developing Natpara® (rhPTH[1-84]) for the treatment of adult hypoparathyroidism and expects to submit its Biologic License Application (BLA) to the FDA in 2013.
NPS's 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, Janssen Pharmaceuticals, Kyowa Hakko Kirin, and Takeda GmbH.
"NPS," "NPS Pharmaceuticals," "Gattex," and "Natpara" 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.
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. Forward looking statements include, but are not limited to, statements concerning our future financial performance. Risks associated to the company's business include, but are not limited to, the risks associated with any failure by the company to successfully commercialize Gattex, including the risk that physicians and patients may not see the advantages of Gattex and may therefore be reluctant to utilize the product, the risk that private and public payers may be reluctant to cover or provide reimbursement for Gattex, the risks associated with the company's strategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect of legislation effecting healthcare reform in the United States, 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, whether as a result of new information, future events or otherwise.