BEDMINSTER, N.J.--(BUSINESS WIRE)--NPS Pharmaceuticals, Inc. (NASDAQ:NPSP) a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today joins patient organizations, healthcare professionals, researchers, public health authorities and the biopharmaceutical industry in recognizing Rare Disease Day. As a “Friend of Rare Disease Day,” NPS strives to increase recognition of rare diseases and educate the public about their global impact on society. Internationally, the observance is led by the European Organisation for Rare Diseases (EURORDIS) and in the US by the National Organization for Rare Disorders (NORD).
“Even though increased interest in orphan drug development and recent regulatory approvals have brought hope to some people living with rare diseases, our job is far from over when you consider the vast number of patients who still have few, if any, treatment options,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “We are proud to stand beside EURORDIS, NORD and their network of advocates in voicing the needs of these patients on Rare Disease Day.”
By definition, a rare disease is one that affects less than 200,000 people in the US and up to 245,000 in Europe. Some rare diseases may affect less than a dozen people, while others near the threshold for the definition. In the US and Europe respectively, an estimated 30 million people are living with a rare disease, which equates to approximately 10 percent of the US population and six percent of Europeans.
Obtaining a diagnosis can be a lengthy and frustrating experience for those living with a rare disease that can take many years. And, once a diagnosis is received, treatment options are not always available. In the US, less than 10 percent of the 6,800 rare diseases that are recognized by the National Institutes of Health have approved treatment options.
“We are looking forward to unprecedented participation for Rare Disease Day 2014, as participants in 80 countries are taking part this year from around the world,” said Yann Le Cam, Chief Executive Officer of EURORDIS. “Individuals and organizations can participate in many ways, including our Raise and Join Your Hands program that promotes solidarity among rare disease patients around the world, signing up as “Friends of Rare Disease Day,” joining us on our social media platforms, or even planning an activity that raises awareness for the observance.”
Rare Disease Day is held annually on the last day of February in an effort to bring recognition to rare diseases as a global health challenge. February 28, 2014, marks the seventh Rare Disease Day. For more information about the observance, visit www.rarediseaseday.org or www.rarediseaseday.us.
EURORDIS is a non-governmental patient-driven alliance of 606 rare disease patient organizations in 56 countries. EURORDIS strives to build a strong pan-European community of patient organizations and people living with rare diseases, to be their voice at the European level, and – directly or indirectly – to fight against the impact of rare diseases on their lives. EURORDIS leads global efforts for Rare Disease Day, which it established in 2008. The observance is celebrated around the world on the last day of February each year and its purpose is to raise awareness of rare diseases as a public health issue. For more information about EURORDIS, visit www.eurordis.org.
Established in 1983, the National Organization for Rare Disorders (NORD) is a unique federation of voluntary health organizations dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. A nonprofit organization, NORD represents the 30 million Americans with rare diseases and is committed to the identification, treatment and cure of rare disorders through patient assistance, education, advocacy, research and patient/family services. For more information about NORD, visit www.rarediseases.org.
About NPS Pharmaceuticals
NPS Pharmaceuticals is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company’s lead product, Gattex® (teduglutide [rDNA origin]) for injection is approved in the US for adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. In the EU, teduglutide (trade name: Revestive®) is approved for the treatment of adult patients with SBS; patients should be stable following a period of intestinal adaptation after surgery. Teduglutide is not approved for the treatment of pediatric SBS patients. The safety and efficacy of teduglutide in this population is currently being evaluated in a global registration trial.
A Biologics License Application is undergoing FDA review for Natpara® (rhPTH [1-84]) for the treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone. The Prescription Drug User Fee Act goal date for the Natpara application is October 24, 2014. NPS’ earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium sensing 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.
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 the company’s future financial performance and plans for the commercialization of its products. 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/Revestive (teduglutide [rDNA origin]) for injection, including the risk that physicians and patients may not see the advantages of Gattex/Revestive 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, risks related to regulatory approvals for Natpara (recombinant human parathyroid hormone 1-84 (rhPTH 1-84)), 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.