NPS Pharmaceutical in Talks with FDA After More Positive Results with Gattex
NPS Pharmaceuticals issued positive results from a phase III extension study of Gattex (teduglutide), an injectable glucagon-like peptide-2 (GLP-2) analog, for the treatment of short bowel syndrome (SBS) in subjects who are dependent upon parenteral nutrition (PN). PN is the practice of feeding a patient intravenously, the cost of which can exceed $100,000 a year.
SBS is a disabling syndrome that can arise after a resection of the small bowel. There are an estimated 10,000 to 15,000 SBS patients in North America who are PN-dependent.
Gattex was granted Orphan Drug status in the U.S. for SBS in August 2000.
This extension study enrolled 65 of the 71 subjects who had completed a 24-week randomized phase III study that evaluated low dose Gattex (0.05 mg/kg/day) and high-dose Gattex (0.10 mg/kg/day) versus placebo. The primary objective of the study was to assess the long-term safety and tolerability of daily Gattex dosing for up to 52 weeks.
Sixty-eight percent of the subjects who had received low-dose Gattex therapy and continued on low-dose Gattex, and 52% of the subjects who had received high-dose Gattex therapy and continued on high-dose Gattex achieved a 20% or greater reduction in PN after a total of 52 weeks of therapy.
Subjects treated with low-dose Gattex showed a mean 51% reduction in PN volume from pretreatment baseline to the end of 52 weeks (p< 0.001) and those treated with high-dose Gattex experienced a mean 24% reduction (p< 0.001).All of the subjects (100%) who had previously received placebo in the phase III study and were randomized to low-dose
Gattex therapy, and two out of seven subjects who had previously received placebo in the phase III study and were randomized to high-dose Gattex therapy, achieved a 20% or greater reduction in PN after a total of 28 weeks of therapy in the extension study.
Treatment was well tolerated in both dose groups with no statistical differences in the rate of adverse events compared with the placebo group.
Based on a series of positive phase III results, NPS is currently meeting with the FDA to discuss the path towards regulatory approval.