The US Food and Drug Administration (FDA) on December 21 approved Gattex (teduglutide), a first-in-class, ultra-orphan therapy for treating adults with short-bowel syndrome (SBS). SBS is a serious disorder in which the body is unable to absorb enough nutrients and fluids through the gastrointestinal tract and so patients must receive them intravenously. In phase 3 studies, Gattex given as daily subcutaneous injections improved intestinal absorption of fluids and nutrients, reducing—and sometimes eliminating—the need for parenteral nutrition. It is a recombinant analog of human glucagon-like peptide 2 (GLP-2), a 33-amino-acid peptide secreted in the gut to enhance absorption, increase vascularization and slow gastric motility. Pharmacological activities of Gattex are limited to the gastrointestinal tract, where most of the GLP-2 receptors are located. Despite a $295,000 annual price tag, 200–300 patients are expected to try Gattex this year and peak sales are anticipated to exceed $350 million, according to Francios Nader, CEO of NPS Pharmaceuticals in Bedminster, New Jersey, which is launching Gattex in the US this quarter. Gattex has been associated with cancer and other serious conditions, so the FDA has approved the drug with a risk evaluation and mitigation strategy. Last year, on August 30, the EU approved Takedas's drug Revestive (teduglutide) for SBS, but the Tokyo-based pharma has not yet launched it in the EU.
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