PARSIPPANY, N.J., Sept. 19 NPS Pharmaceuticals,Inc. (Nasdaq: NPSP) announced today that the U.S. Food and DrugAdministration's Office of Orphan Products Development has granted orphan drugdesignation to PREOS(R) (parathyroid hormone [rDNA origin] for injection) forthe treatment of hypoparathyroidism, a rare deficiency of parathyroid hormonefor which there is no FDA-approved therapy.
Hypoparathyroidism afflicts approximately 65,000 people in the UnitedStates and an estimated 120,000 outside of the U.S. It is most often causedby injury to or removal of the parathyroid glands during neck surgery. It alsooccurs as an autoimmune disorder associated with HIV infection. Currenttreatments include vitamin D analogs and large doses of supplemental calciumto relieve the symptoms of neuromuscular irritability associated withhypoparathyroidism. Despite these palliative therapies, the absence ofendogenous parathyroid hormone compromises the body's ability to properlyabsorb calcium. This invariably results in hypercalciuria andnephrocalcinosis, a serious condition characterized by calcium deposition inthe kidneys. PREOS could potentially be a first-in-class therapy for use inthis condition.
"We greatly appreciate the FDA's support of our efforts to evaluate theuse of PREOS as a hormone replacement therapy to treat hypoparathyroidism. Theresults of the proof-of-concept study using PREOS in this indication have beenvery encouraging, especially when compared to current palliative therapiesthat often result in long-term toxicities and complications for patients. Weare excited to expand the development of PREOS beyond our initial focus onosteoporosis to include the drug's potential use as a treatment forhypoparathyroidism," said Dr. Francois Nader, executive vice president andchief operating officer of NPS.
Orphan status is granted by the FDA to promote the development of productsthat demonstrate promise for the treatment of rare diseases affecting fewerthan 200,000 Americans annually. Orphan drug designation entitles NPS to aseven-year period of marketing exclusivity in the United States for PREOS, ifit is approved by the FDA for the treatment of hypoparathyroidism, and enablesthe company to apply for research funding, tax credits for certain researchexpenses, and a waiver from the FDA's application user fee.
NPS Pharmaceuticals is a biopharmaceutical company focused on thedevelopment and commercialization of small molecules and recombinant proteinsas drugs, primarily for the treatment of metabolic, bone and mineral, andcentral nervous system disorders. The company has drug candidates in variousstages of clinical development. Additional information is available on thecompany's website, http://www.npsp.com.
PREOS is recombinant human parathyroid hormone (PTH). NPS has studiedPREOS in a number of clinical settings to document its safety and effects onbone. The pivotal Phase 3 study, known as TOP (Treatment of Osteoporosis withPTH), was a multi-center, randomized, double-blind and placebo-controlledclinical trial designed to evaluate the potential of PTH to reduce the risk offirst and subsequent vertebral fractures in post-menopausal women.
In the TOP study, PREOS demonstrated a statistically significant reductionin the risk of new vertebral fractures in women with and without pre-existingosteoporosis-related fractures. Results from the TOP study have been thefoundation of both the E.U. and the U.S. marketing authorization applications.
PREOS has been approved in Europe under the brand name Preotact and isbeing marketed by Nycomed.
Note: Statements made in this press release, which are not historical innature, constitute forward-looking statements for purposes of the safe harborprovided by the Private Securities Litigation Reform Act of 1995. Thesestatements are based on managemen