The US Food and Drug Administration’s (FDA) Gastrointestinal Drugs Advisory Committee voted unanimously to recommend approval of NPS Pharmaceuticals' 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 per cent 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 favourable benefit-risk profile of Gattex,” said Francois Nader, “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 per cent 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.
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.
NPS Pharmaceuticals is a biopharmaceutical company focused on bringing orphan products to patients with rare disorders and few, if any, therapeutic options.