FDA Approves Extension of Treatment for Short Bowel Syndrome to Include Children

Officials with the FDA have approved extending the indication of Takeda Pharmaceuticals’ teduglutide (Gattex) for injection to pediatric patients age 1 year and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous (IV) feeding, according to the drug manufacturer.

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 patient’s ability to absorb nutrients and reduce long-term dependence on parenteral support (PS).

“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, MD, Medical Director of Intestinal Rehabilitation and Nutrition Support, Children’s Hospital Los Angeles, in a prepared statement. “I’m pleased that patients have access to a medication that may help them reach that goal.”

According to Takeda, teduglutide is the first and only medicine that mimics naturally occurring glucagon-like peptide-2 (GLP-2). The recommended dosage of GATTEX for both adults and pediatric patients is 0.05 mg/kg once daily by subcutaneous injection. Use of the GATTEX 5 mg kit is not recommended in pediatric patients weighing less than 10 kg.

In a pharmacodynamic study in adults, teduglutide was shown to improve the amount of fluids absorbed by the intestines.

In a 24-week pediatric study, teduglutide helped reduce the volume of daily PS required and time spent administering PS. Some children achieved complete freedom from PS. Fifty- nine pediatric patients ages 1-17 years with SBS chose whether to receive teduglutide or standard of care (SOC). Patients who chose to receive teduglutide treatment were subsequently randomized in a double-blind manner to 0.025 mg/kg/day (n=24) or 0.05 mg/kg/day (n=26), while 9 patients enrolled in the SOC arm.

At the end of the 24-week study, 69% of patients who took teduglutide 0.05 mg/kg each day reduced PS volume by 20% or more. Based on patient-diary data, patients who received teduglutide 0.05 mg/kg/day experienced a 42% mean reduction in PS volume (mL/kg/day) from baseline (-23 mL/kg/day from baseline).

At week 24, 38% of patients (10/26) were able to reduce PS infusion by at least 1 day per week. Patients reduced their PS infusion time by 3 hours per day on average compared to baseline. In addition, during this study 12% children who received teduglutide 0.05 mg/kg/day completely weaned off PS.

Teduglutide has a demonstrated safety profile that is similar overall in pediatric and adult patients. The most common adverse reactions (≥10%) seen in adult patients treated with teduglutide in clinical trials were abdominal pain, nausea, upper respiratory tract infection, abdominal distension, injection site reaction, vomiting, fluid overload, and hypersensitivity.

Malabsorption of fluids and nutrients can place children with SBS at risk for diarrhea, dehydration, electrolyte disturbances, and malnutrition. Parents and caregivers of children with SBS who are dependent on PS have reported in studies that they experience deterioration in overall quality of life. PS reductions may benefit children with SBS, and potentially give them more freedom for daily-life activities.

“Addressing high unmet needs of patients with complex and debilitating gastrointestinal (GI) conditions is a focus of Takeda’s work,” said Andrew Grimm, Global Clinical Development Lead, Takeda, in a prepared statement. “As the first US-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.”

Reference

U.S. FDA Approves GATTEX® (teduglutide) for Children 1 Year of Age and Older With Short Bowel Syndrome (SBS) [news release]. Lexington, MA; May 17, 2019: Takeda Pharmaceuticals. https://www.prnewswire.com/news-releases/us-fda-approves-gattex-teduglutide-for-children-1-year-of-age-and-older-with-short-bowel-syndrome-sbs-300852294.html?tc=eml_cleartime. Accessed May 17, 2019.