The US Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) action date for NPS Pharmaceuticals' New Drug Application (NDA) for Gattex (teduglutide) by three months to December 30, 2012.
The FDA extended the action date to provide time for a full review of the submission; such extensions are provided for by regulation when information is submitted within three months of a PDUFA action date.
Gattex is a novel, recombinant analog of human glucagon-like peptide 2, a peptide involved in normal intestinal function and fluid and nutrient absorption. The proposed indication for Gattex is the treatment of adult patients with short bowel syndrome. The company completed its NDA submission for Gattex in November 2011.
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 short bowel syndrome 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 short bowel syndrome patients, it does not improve the body's own ability to absorb nutrients. PN itself 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 a poor quality of life with difficulty sleeping, frequent urination and loss of independence.
Gattex (teduglutide) is a novel, recombinant analogue 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). Two phase III studies of teduglutide demonstrated a favorable safety profile and significant reductions in mean PN volume from baseline to end of treatment. In addition some patients were weaned off PN entirely.
Gattex has received orphan drug designation for the treatment of SBS from the European Medicines Agency (EMA) and the FDA.
NPS retains all rights to Gattex in North America, and has granted Nycomed the rights to develop and commercialize teduglutide outside North America and Israel. In June 2012, the Committee for Medicinal Products for Human Use (CHMP) of the EMA adopted a positive opinion, recommending the granting of a marketing authorization for teduglutide (trade name in Europe: Revestive) as a once-daily treatment for adult patients with short bowel syndrome.
NPS Pharmaceuticals is focused on bringing orphan products to patients with rare disorders and few, if any, therapeutic options.