BEDMINSTER, N.J.--NPS Pharmaceuticals, Inc. (NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today reported that the U.S. Food and Drug Administration (FDA) has extended the company's Prescription Drug User Fee Act (PDUFA) action date for its Natpara Biologics License Application (BLA) by three months from October 24, 2014 to January 24, 2015. The FDA extended the action date to provide time for a full review of a major amendment; such extensions are provided for by regulation. In addition, the FDA requested that NPS Pharma submit a Risk Evaluation and Mitigation Strategy (REMS) for Natpara and the company is working to finalize the REMS in advance of the revised PDUFA action date. To date, the FDA has not requested that additional clinical studies be completed prior to the approval of Natpara; however, the company expects a post-approval study commitment.
"We continue to work closely with the FDA to finalize the review of our BLA for Natpara and appreciate the effective collaboration with the Agency to date," said Francois Nader, MD, president and chief executive officer of NPS Pharma. "We continue to advance our commercial readiness activities and are planning for the launch of Natpara in the second quarter of 2015."
Natpara is a bioengineered replacement therapy for endogenous parathyroid hormone (PTH) that NPS Pharma has developed for the treatment of Hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of PTH.
About Hypoparathyroidism
Hypoparathyroidism is a rare endocrine disorder affecting only approximately 50,000 people in the U.S. in which the parathyroid glands fail to produce sufficient amounts of PTH or where the hormone lacks biologic activity. PTH plays a central role in a variety of critical physiological functions in the body. Insufficient levels of PTH lead to many physiological abnormalities, including low levels of calcium and high levels of phosphate in the blood, and an inability to convert native vitamin D into its active state, which helps the body properly absorb oral calcium.
With no approved replacement therapy, the only option available is using large doses of oral calcium and active vitamin D to try and manage symptoms of hypocalcemia by increasing serum calcium, but this does not address the underlying cause of the disorder. In addition, the administration of this regimen does not enable the activation of native vitamin D, and is often difficult due to calcium fluctuations and the challenges of balancing symptom control while minimizing the amount of excess calcium being filtered through the kidneys. The long-term use of calcium and vitamin D may lead to serious complications, because increasing serum calcium without decreasing serum phosphate may lead to irreversible calcium-phosphate deposits in the kidneys, heart or brain.
About NPS Pharma
NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company's current therapeutic areas of focus are gastrointestinal disease and endocrine disorders. These include Short Bowel Syndrome, a potentially fatal gastrointestinal disorder in which patients may have to rely on parenteral nutrition for their survival; Hypoparathyroidism, a complex endocrine disorder in which the parathyroid glands are either absent or damaged, and the body produces insufficient or no parathyroid hormone; and Autosomal Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium homeostasis caused by mutations of the calcium-sensing receptor gene. NPS Pharma continues to seek in-licensing opportunities to develop new therapies for a broad range of rare diseases, and 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. NPS Pharma has operations in the U.S., Canada, Europe, Latin America and Japan. Learn more at: www.npsp.com
"NPS Pharma" and "NPS Pharmaceuticals" are the company's trademarks.
Disclosure notice
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, including statements regarding launch timelines and plans for Natpara, beliefs or expectations regarding our products in development, statements concerning the company's plans for international expansion, beliefs or expectations regarding potential revenue and earnings from product sales, including beliefs regarding our ability to grow sales, expectations regarding the market size for our products, including those in development, and beliefs or expectations regarding our operating expenses. 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 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 press release and NPS undertakes no duty to update this information, whether as a result of new information, future events or otherwise.