NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a specialty pharmaceutical company developing orphan therapeutics for rare gastrointestinal and endocrine disorders, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed for review the company’s New Drug Application (NDA) for GATTEX® (teduglutide) for the treatment of adults with short bowel syndrome or SBS. SBS is a rare disorder characterized by inadequate absorption of fluids and nutrients in people who have had a significant portion of their small intestine surgically removed due to disease or injury. GATTEX is a novel, recombinant analog of human glucagon-like peptide 2, a peptide involved in gastrointestinal regeneration and repair. The acceptance of the GATTEX NDA is the FDA’s determination that the application is sufficiently complete to permit a substantive review.
“NPS has met yet another milestone in building NPS as a premier orphan drug company,” said Francois Nader, M.D., president and chief executive officer of NPS Pharmaceuticals. “We appreciate the FDA’s acceptance of the Gattex marketing application as we believe that GATTEX has the potential to make a meaningful difference in the lives of patients suffering from SBS by reducing or in some cases eliminating their dependence on parenteral nutrition and intravenous fluids.”
Under the Prescription Drug User Fee Act (PDUFA), the FDA will notify NPS of its action date, the date FDA commits to providing a decision on the NDA, within the Filing Review Notification, also referred to as the Day 74 letter.
The company’s SBS clinical development program represents the largest and most comprehensive to date. The information in the NDA is derived from 14 completed and one ongoing clinical study. A total of 566 subjects have been treated with teduglutide. Of the 566 subjects treated with teduglutide, 299 subjects were treated in the clinical pharmacology studies, 94 subjects in Crohn’s Disease studies, and 173 subjects in the SBS efficacy and safety studies. The initial NDA included data from 75 SBS subjects who had at least 12 months of exposure to GATTEX.
About Short Bowel Syndrome
Short bowel syndrome, or SBS, is a highly disabling condition that can lead to serious life-threatening complications and impair a patient's quality-of-life. SBS typically arises after extensive resection of the bowel due to Crohn's disease, ischemia or other conditions. SBS is associated with intestinal failure and the inability to absorb sufficient nutrients and/or fluids through the gastrointestinal tract. Patients with SBS are highly prone to malnutrition, diarrhea, dehydration, and an inability to maintain weight due to the reduced intestinal capacity to absorb macronutrients, water, and electrolytes. As a result, many patients require the chronic use of parenteral nutrition (PN) and intravenous (IV) fluids to supplement their nutritional needs and stabilize their hydration. PN/IV fluids bypass the digestive tract and are delivered directly into the bloodstream through a central venous catheter. Although PN/IV can meet basic nutrition and fluid requirements, it does not improve the body's ability to absorb nutrients.
The long-term use of PN/IV fluids is associated with shortened life span and life-threatening complications such as blood clots and liver damage. The development of PN-associated liver disease predisposes patients to an increased incidence of sepsis, increased mortality rates, and the potential to develop irreversible liver damage. Patients on parenteral support often experience a poor quality of life with difficulty sleeping, frequent urination, and loss of independence.
There are an estimated 10,000 to 15,000 SBS patients in the U.S. who are dependent on parenteral nutrition and intravenous fluids.
About NPS Pharmaceuticals
NPS Pharmaceuticals is a biopharmaceutical company focused on bringing orphan products to patients with rare disorders and few, if any, therapeutic options. NPS is advancing two late-stage registration programs. A New Drug Application is undergoing FDA review for GATTEX® (teduglutide) as a treatment for adult short bowel syndrome (SBS) and a Phase 3 registration study has been completed for NATPARA™ (recombinant human parathyroid hormone (rhPTH [1-84]) in adult hypoparathyroidism. NPS’ earlier stage pipeline includes two calcilytic compounds, NPSP790 and NPSP795, with potential application in rare disorders involving increased calcium receptor activity, such as autosomal dominant hypocalcemia with hypercalciuria (ADHH). NPS complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Kyowa Hakko Kirin, Nycomed, and Ortho-McNeil Pharmaceutical.
“NPS,” “NPS Pharmaceuticals,” and “GATTEX” are the company’s registered trademarks. All other trademarks, trade names or service marks appearing in this press release are the property of their respective owners.
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. Risks associated to the company’s business include, but are not limited to, the risks associated with any failure by the company to successfully complete its preclinical and clinical studies within the projected time frames or not at all, the risk of not gaining marketing approvals for GATTEX and NATPARA, the risks associated with the company’s strategy, 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.