NPS Pharmaceuticals PREOS Granted U.S. Orphan Drug Status for Treatment of Hypoparathyroidism
PARSIPPANY, N.J., Sept. 19 -- NPS Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration's Office of Orphan Products Development has granted orphan drug designation to PREOS(R) (parathyroid hormone [rDNA origin] for injection) for the treatment of hypoparathyroidism, a rare deficiency of parathyroid hormone for which there is no FDA-approved therapy.
Hypoparathyroidism afflicts approximately 65,000 people in the United States and an estimated 120,000 outside of the U.S. It is most often caused by injury to or removal of the parathyroid glands during neck surgery. It also occurs as an autoimmune disorder associated with HIV infection. Current treatments include vitamin D analogs and large doses of supplemental calcium to relieve the symptoms of neuromuscular irritability associated with hypoparathyroidism. Despite these palliative therapies, the absence of endogenous parathyroid hormone compromises the body's ability to properly absorb calcium. This invariably results in hypercalciuria and nephrocalcinosis, a serious condition characterized by calcium deposition in the kidneys. PREOS could potentially be a first-in-class therapy for use in this condition.
"We greatly appreciate the FDA's support of our efforts to evaluate the use of PREOS as a hormone replacement therapy to treat hypoparathyroidism. The results of the proof-of-concept study using PREOS in this indication have been very encouraging, especially when compared to current palliative therapies that often result in long-term toxicities and complications for patients. We are excited to expand the development of PREOS beyond our initial focus on osteoporosis to include the drug's potential use as a treatment for hypoparathyroidism," said Dr. Francois Nader, executive vice president and chief operating officer of NPS.
Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. Orphan drug designation entitles NPS to a seven-year period of marketing exclusivity in the United States for PREOS, if it is approved by the FDA for the treatment of hypoparathyroidism, and enables the company to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA's application user fee.
NPS Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of small molecules and recombinant proteins as drugs, primarily for the treatment of metabolic, bone and mineral, and central nervous system disorders. The company has drug candidates in various stages of clinical development. Additional information is available on the company's website, http://www.npsp.com.
PREOS is recombinant human parathyroid hormone (PTH). NPS has studied PREOS in a number of clinical settings to document its safety and effects on bone. The pivotal Phase 3 study, known as TOP (Treatment of Osteoporosis with PTH), was a multi-center, randomized, double-blind and placebo-controlled clinical trial designed to evaluate the potential of PTH to reduce the risk of first and subsequent vertebral fractures in post-menopausal women.
In the TOP study, PREOS demonstrated a statistically significant reduction in the risk of new vertebral fractures in women with and without pre-existing osteoporosis-related fractures. Results from the TOP study have been the foundation of both the E.U. and the U.S. marketing authorization applications.
PREOS has been approved in Europe under the brand name Preotact and is being marketed by Nycomed.
Note: 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 management'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. All information in this press release is as of September 19, 2007 and we undertake no duty to update this information. A more complete description of these risks can be found in our filings with the Securities and Exchange Commission, including our Annual Report on Form 10-K for the year-ended December 31, 2006 and our Quarterly Report on Form 10-Q for the quarter-ended June 30, 2007.