FDA Expands Use of Gattex to Include Short Bowel Syndrome
May 20, 2019
The U.S. Food and Drug Administration has approved extending the indication of Takeda Pharmaceuticals’ Gattex to pediatric patients one year of age and older with short bowel syndrome (SBS) who need additional nutrition or fluids from intravenous.
The agency first approved Gattex in 2012 for use in adults with the disorder, often due to surgery that removed a large part of the small intestine.
In children, short bowel syndrome is a life-threatening, chronic, and rare malabsorption disorder resulting from surgical removal of a large portion of the intestine, which is typically due to congenital or acquired conditions of the newborn or trauma. Children with SBS are unable to absorb enough nutrients and fluids from what they eat and drink alone. A goal of SBS treatment is to restore the remaining intestine’s ability to absorb nutrients and reduce long-term dependence on intravenous nutritional support referred to as parenteral support.
Gattex is a recombinant analog of human glucagon-like peptide-2. It mimics 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.
“As a pediatric gastroenterologist, one of my main treatment goals for children with SBS is to reduce their dependency on parenteral support,” said Beth Carter, medical director of Intestinal Rehabilitation and Nutrition Support, Children’s Hospital Los Angeles. “I’m pleased that patients have access to a medication that may help them reach that goal.”
In a 24-week pediatric study, Gattex helped reduce the volume of daily parenteral support (PS) required and time spent administering PS. Some children even achieved complete freedom from PS. A total of 59 pediatric patients with SBS aged one year through 17 years chose whether to receive Gattex or standard of care. Patients who chose to receive Gattex treatment were subsequently randomized in a double-blind manner to two different doses, while nine patients enrolled in the standard of care arm.
At the end of the 24-week study, 69 percent of patients in the higher dosing arm reduced PS volume by 20 percent or more. Based on patient-diary data, this cohort experienced a 42 percent mean reduction in PS volume from baseline. At week 24, 38 percent of patients were able to reduce PS infusion by at least one day per week. Patients reduced their PS infusion time by three hours per day on average compared to baseline. In addition, during this study three out of 26 children who received Gattex completely weaned off PS. Gattex’s safety profile is similar overall in pediatric and adult patients.
“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,” said Andrew Grimm, global clinical development lead at Takeda.
Author: Rare Daily Staff
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