NPS Pharmaceuticals Announces FDA Acceptance Of Supplemental New Drug Application For Gattex® (Teduglutide [rDNA Origin]) For Injection

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Supplemental New Drug Application (sNDA) for Gattex ® (teduglutide [rDNA origin]) for injection. In the U.S., Gattex is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. The company is seeking additions to the product label for long-term data from STEPS 2, a two-year open-label extension study in which 88 adult patients with SBS dependent on parenteral support received Gattex 0.05 mg/kg/day.

Acceptance of the sNDA indicates that the application is sufficiently complete to permit a substantive review. The application has been assigned a Prescription Drug User Fee Act (PDUFA) date of June 28, 2014.

“We are pleased that the FDA has accepted our sNDA for review,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals. “The data from STEPS 2 we’re proposing to include in the label show that patients on Gattex treatment beyond one year continue to achieve clinically meaningful reductions in parenteral support requirements, with some achieving complete independence. This is encouraging for patients with Short Bowel Syndrome, as the ability to reduce or even completely eliminate the need for parenteral support could meaningfully impact their lives.”

About Gattex® (teduglutide [rDNA origin]) for Injection

Gattex® (teduglutide [rDNA origin]) for injection for subcutaneous use is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. Gattex is indicated for the treatment of adult patients with Short Bowel Syndrome (SBS) who are dependent on parenteral support. Significant reductions in mean PN/IV infusion volume from baseline to end of treatment were seen in the Phase 3 studies of Gattex. In addition, some patients were able to achieve independence from PN/IV support during these trials. The most common side effects of Gattex include stomach area (abdomen) pain or swelling, skin reaction where the injection was given, nausea, headache, cold or flu like symptoms, vomiting, and holding too much fluid in the body (swelling of face, ankles, hands or feet).

The European Commission granted European market authorization on August 30, 2012 for the medicinal product teduglutide (trade name in Europe: Revestive®) as a once-daily treatment for adult patients with SBS. NPS is preparing to engage in the pricing and reimbursement processes in key European countries with the goal of commercially launching Revestive in Europe in the first half of 2014.

Teduglutide has orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.

Important Safety Information (ISI)

Gattex has been associated with serious risks including:

Neoplastic growth. There is a risk for acceleration of neoplastic growth. Colonoscopy of the entire colon with removal of polyps should be done before initiating treatment with Gattex and is recommended after 1 year. Subsequent colonoscopies should be done as needed, but no less frequently than every 5 years. In case of intestinal malignancy discontinue Gattex. The clinical decision to continue Gattex in patients with active non-gastrointestinal malignancy should be made based on risk and benefit considerations.

Intestinal obstruction. In patients who develop obstruction, Gattex should be temporarily discontinued pending further clinical evaluation and management.

Biliary and pancreatic disease. Patients should undergo laboratory assessment (bilirubin, alkaline phosphatase, lipase, amylase) before starting Gattex. Subsequent laboratory tests should be done every 6 months. If clinically meaningful changes are seen, further evaluation is recommended including imaging, and continued treatment with Gattex should be reassessed.

Fluid overload. There is a potential for fluid overload while on Gattex. If fluid overload occurs, especially in patients with cardiovascular disease, parenteral support should be appropriately adjusted, and Gattex treatment reassessed.

Prescribers should select the appropriate patients to receive Gattex in accordance with the approved prescribing information, discuss the benefits and risks of Gattex with patients, and monitor patients as specified in the approved prescribing information and report adverse events to NPS' Gattex information line at 1-855-5GATTEX (1-855-542-8839) or event/product complaint line at 1-855-215-5550.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide. The company’s lead product, Gattex® (U.S.)/Revestive® (EU) (teduglutide [rDNA origin]) for injection is approved for adult Short Bowel Syndrome (SBS) patients who are dependent on parenteral support. NPS has also developed Natpara® (rhPTH [1-84]) for the treatment of hypoparathyroidism and submitted its Biologic License Application to the U.S. Food and Drug Administration in October 2013.

NPS’ earlier stage pipeline includes NPSP795, a calcilytic compound with potential application in rare disorders involving increased calcium 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. Additional information about NPS is available through its corporate website, http://www.npsp.com.

“NPS,” “NPS Pharmaceuticals,” “Gattex,” “Natpara,” “Preotact,” and “Revestive” 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. 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 (teduglutide [rDNA origin]) for injection, including the risk that physicians and patients may not see the advantages of Gattex 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.

Copyright Business Wire 2010

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