FDA Advisory Committee Unanimously Recommends Approval of Gattex® (teduglutide) for Adults with Shor
FDA Advisory Committee Unanimously Recommends Approval of Gattex® (teduglutide) for Adults with Short Bowel Syndrome (SBS)
-- Company also reports additional five patients achieved independence from PN/IV fluids in ongoing STEPS 2 study --
-- NPS to host conference call tomorrow at 8:30 AM ET --
BEDMINSTER, N.J.--(BUSINESS WIRE)-- NPS Pharmaceuticals, Inc. (NAS: NPSP) , a biopharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, announced today that the U.S. Food and Drug Administration's (FDA) Gastrointestinal Drugs Advisory Committee voted unanimously to recommend approval of Gattex® (teduglutide) for adults with short bowel syndrome (SBS). The committee's recommendation will be considered by the FDA in its review of the company's New Drug Application (NDA) for Gattex. The Prescription Drug User Fee Act (PDUFA) date for completion of the review is December 30, 2012.
NPS also today reported that five additional patients have successfully achieved independence from parenteral nutrition (PN) and intravenous (IV) fluids while on long-term Gattex® (teduglutide) therapy in STEPS 2, a 24-month open-label study in adult SBS. To date, 12 patients or 14 percent have achieved independence from PN/IV fluids while on Gattex therapy in the ongoing STEPS 2 study.
"The results of today's advisory committee reinforced our belief in the favorable benefit-risk profile of Gattex," said Francois Nader, MD. "The unique mechanism of Gattex could make it the first and only long-term therapy that treats the underlying cause of this rare disorder by helping patients increase their ability to absorb nutrients and fluids to reduce or even eliminate their dependence on parenteral support. With 14 percent of patients completely eliminating the need for parenteral support in STEPS 2, we believe Gattex could be a life-changing therapy for patients with short bowel syndrome."
SBS is a serious, complex disorder in which the body is unable to absorb enough nutrients and fluids through the gastrointestinal track to sustain life. SBS is typically treated with PN/IV fluids that do not treat the underlying disorder, which is the body's inability to absorb nutrients and fluids. In addition, long-term use of PN/ IV fluids can be associated with life-threatening complications such as liver damage, serious bloodstream infections, and blood clots.
Gattex is a novel, recombinant analog of human glucagon-like peptide 2 (GLP-2), a peptide involved in normal intestinal function and fluid and nutrient absorption. If approved, Gattex would be the first FDA-approved therapy indicated for the long-term treatment of adults with SBS. The company's SBS clinical development program represents the largest and most comprehensive to date. The advisory committee reviewed data from the program, including information in the Gattex NDA, which 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 Gattex, 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. Of the 566 Gattex-treated subjects, 97 SBS subjects had at least 12 months of exposure to Gattex. Across the company's Phase 3 studies, a total of 15 patients were able to achieve independence from PN/IV while on 0.05 mg/kg/day Gattex.
Conference Call Information
NPS will host a conference call beginning tomorrow, Wednesday, October 17, at 8:30 a.m. Eastern Time. To participate in the conference call, dial (866) 356-4279 and use pass code 96377660. International callers may dial (617) 597-5394, using the same pass code. In addition, a live audio of the conference call will be available over the Internet. Interested parties can access the event through the NPS website, http://www.npsp.com.
For those unable to participate in the live call, a replay will be available at (888) 286-8010, with pass code 14044215, until midnight Eastern Time, October 31, 2012. International callers may access the replay by dialing (617) 801-6888, using the same pass code. The webcast will also be available through the NPS website for the same period.
About Short Bowel Syndrome
Short bowel syndrome (SBS) is a highly disabling condition that can impair a patient's quality of life and lead to serious life-threatening complications. SBS typically arises after extensive resection of the bowel due to Crohn's disease, ischemia or other conditions. SBS patients often suffer from malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight loss due to the reduced intestinal capacity to absorb nutrients, water, and electrolytes. The usual treatment for SBS is nutritional support, including parenteral nutrition (PN) and/or intravenous (IV) fluids to supplement and stabilize nutritional needs.
Although PN can provide nutritional support for SBS patients, it does not improve the body's own ability to absorb nutrients. PN is associated with serious complications, such as infections, blood clots or liver damage, and the risks increase the longer patients are on PN. Patients on PN often experience poor quality of life with difficulty sleeping, and frequent urination, and patients receiving chronic PN often experience a loss of independence.
Gattex (teduglutide) is a novel, recombinant analog of human glucagon-like peptide 2, a protein involved in the rehabilitation of the intestinal lining. It has been developed to reduce dependence on parenteral nutrition (PN) in adult patients with short bowel syndrome (SBS). Significant reductions in mean PN/IV infusion volume from baseline to end of treatment were seen in the Phase 3 studies of teduglutide. In addition, some patients were able to be weaned off PN during these trials. The most common treatment-emergent adverse events with Gattex in the placebo-controlled studies that occurred at a higher frequency with Gattex were abdominal pain, upper respiratory tract infections, nausea, injection site reactions, abdominal distension, headaches, and gastrointestinal stoma complications.
Gattex has received orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.
In 2007, NPS granted Nycomed, a Takeda company, the rights to develop and commercialize teduglutide outside the United States, Canada, Mexico and Israel. NPS retains all rights to teduglutide in North America. 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 short bowel syndrome.
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 adults with short bowel syndrome (SBS) and a Phase 3 registration study has been completed for Natpara® (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, Janssen Pharmaceuticals, Kyowa Hakko Kirin, and Nycomed, a Takeda company.
"NPS," "NPS Pharmaceuticals," "Gattex," and "Natpara" are the company's 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.
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