European Medicines Agency Validates Marketing Authorization Application for Natpar® (parathyroid hormone (rDNA)) in Hypoparathyroidism

BEDMINSTER, N.J.--()--NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today announced that the European Medicines Agency (EMA) has validated and initiated its review of the company’s marketing authorization application (MAA) for Natpar® (parathyroid hormone (rDNA)) for the treatment of Hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH). If approved, Natpar would be the first and only PTH replacement therapy in Europe for patients with Hypoparathyroidism.

“MAA validation for Natpar is an important achievement for our emerging global endocrine franchise,” said Francois Nader, MD, president and chief executive officer of NPS Pharma. “Without an approved replacement therapy, the current goal of Hypoparathyroidism management is limited to symptom control, which does not address the underlying cause of the disorder. In clinical studies, Natpar has demonstrated similar physiologic effects to the native parathyroid hormone, showing promise as long-term PTH replacement therapy.”

The MAA includes results from 13 pharmacology studies and four company-sponsored efficacy and safety studies. The pivotal Phase 3 study, known as REPLACE, was a multinational, randomized, double-blind, placebo controlled study and the largest clinical trial conducted to date in Hypoparathyroidism. Key findings from the study were published in The Lancet Diabetes and Endocrinology (2013; 1:275-283).

The EMA granted orphan drug designation for Natpar for the treatment of Hypoparathyroidism in January 2014. The U.S. Food and Drug Administration (FDA) granted orphan drug status for the product for the treatment of Hypoparathyroidism in 2007 and a Biologics License Application to market it under the trade name Natpara® is currently under review by the FDA with a Prescription Drug User Fee Act action date of January 24, 2015.

About Hypoparathyroidism

Hypoparathyroidism is a rare condition in which the parathyroid glands fail to produce sufficient amounts of parathyroid hormone (PTH) or where PTH lacks biologic activity. PTH plays a central role in a variety of critical physiological functions in the body. In patients with Hypoparathyroidism, insufficient levels of PTH lead to many physiological abnormalities, including low serum calcium and an inability to convert native vitamin D into its active state to properly absorb dietary calcium.

Acute symptoms of Hypoparathyroidism are largely due to low serum calcium and range from muscle pain and tingling, to lack of focus or ability to concentrate, and anxiety and depression. In extreme cases, life-threatening events, such as arrhythmias and seizures, may occur. In the absence of an approved parathyroid replacement therapy, the standard approach focuses on using large doses of calcium and active vitamin D to increase calcium levels in the blood and reduce the severity of symptoms. However, balancing the administration of large doses of calcium and vitamin D is challenging due to calcium fluctuations and the long-term use of this regimen may lead to serious complications. In addition, calcium and vitamin D do not correct the abnormal bone metabolism due to PTH deficiency or enable the activation of vitamin D.

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 rare 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, 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.

Contacts

NPS Pharmaceuticals, Inc.
Media
Scott Santiamo, +1 908-450-5599
SSantiamo@npsp.com
or
Investors
Susan Mesco, +1 908-450-5516
SMesco@npsp.com

Contacts

NPS Pharmaceuticals, Inc.
Media
Scott Santiamo, +1 908-450-5599
SSantiamo@npsp.com
or
Investors
Susan Mesco, +1 908-450-5516
SMesco@npsp.com