Takeda’s Gattex won approval for a new indication from the U.S. Food and Drug Administration (FDA). For Gattex. Last week, Takeda Pharmaceuticals USA said the FDA approved Gattex (teduglutide) for pediatric patients one year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous (IV) feeding.
For children, SBS is a rare and life-threatening malabsorption disorder resulting from surgical removal of a large portion of the intestine. The segment of intestine removed is typically associated with a congenital condition in a newborn. Children with SBS are unable to absorb enough nutrients and fluids from what they eat and drink alone. The goal for treatment of SBS is to restore the intestine’s ability to absorb nutrients and reduce long-term dependency on parenteral support (PS).
The approval of Gattex marks a first in the U.S. for pediatric SBS patients who depend on parenteral support, Andrew Grimm, global clinical development lead at Takeda, said in a statement. Grimm added that addressing high unmet needs of patients with complex and debilitating gastrointestinal (GI) conditions is a key focus for Takeda.
“As the first U.S.-approved therapy in pediatric SBS patients dependent on PS that improves absorption, Gattex offers these patients new hope to reduce PS requirements and the potential for PS independence. This approval underscores Takeda’s commitment to patients with rare and devastating GI conditions like SBS,” Grimm said in a statement.
“As a pediatric gastroenterologist, one of my main treatment goals for children with SBS is to reduce their dependency on parenteral support,” Beth Carter, medical director of Intestinal Rehabilitation and Nutrition Support at Children’s Hospital Los Angeles, said in a statement. “I’m pleased that patients have access to a medication that may help them reach that goal.”
Gattex, which is also approved for adults with SBS, is designed to mimic naturally occurring glucagon-like peptide-2 (GLP-2), which helps the remaining intestine absorb more nutrients. In a pharmacodynamic study in adults, Gattex was shown to improve the amount of fluids absorbed by the intestines. In the juveniles, Gattex was shown to reduce the volume of daily PS required, as well as time spent administering PS, Takeda said. During the trial, 69 percent of patients who took Gattex daily reduced PS volume by 20 percent or more. After 24 weeks, 38 percent of patients were able to reduce PS infusions by one day per week. Patients reduced their PS infusion time by 3 hours per day on average compared to baseline. Some children even achieved complete freedom from PS, Takeda noted.