The US Food and Drug Administration (FDA) has extended the indication for teduglutide (Gattex, Takeda) injection to children as young as 1 year old with short bowel syndrome (SBS) who need additional intravenous parenteral support, the company has announced.
Teduglutide is the only glucagon-like peptide-2 (GLP-2) analog approved that improves intestinal absorption in patients with SBS who are dependent on parenteral support, the company said today in a news release. It was approved for adults with SBS in 2012.
SBS results from the partial or complete surgical removal of a large portion of the intestine, which is typically due to congenital or acquired conditions of the newborn, or trauma. Extensive loss of the small intestine can lead to poor absorption of fluids and nutrients from food. A goal of SBS treatment is to restore the remaining intestine's ability to absorb nutrients and reduce long-term dependence on parenteral support.
Teduglutide helps improve intestinal absorption of fluids and nutrients, reducing the frequency and volume of parenteral nutrition. The recommended dosage of teduglutide for both adults and pediatric patients is 0.05 mg/kg once daily by subcutaneous injection.
"As a pediatric gastroenterologist, one of my main treatment goals for children with SBS is to reduce their dependency on parenteral support. I'm pleased that patients have access to a medication that may help them reach that goal," Beth Carter, MD, medical director of intestinal rehabilitation and nutrition support at Children's Hospital in Los Angeles, California, said in the release.
Teduglutide was studied in 59 children with SBS ages 1 to 17 years who chose to take the drug or receive standard care. Patients who chose to receive teduglutide treatment were subsequently randomly assigned to 0.025 mg/kg/day (n = 24) or 0.05 mg/kg/day (n = 26); nine patients received standard care.
At the end of the 24-week study, 69% of patients who took teduglutide 0.05 mg/kg daily reduced parenteral support volume by 20% or more. Based on patient-diary data, patients who received this dose of the drug experienced a 42% mean reduction in parenteral support volume (mL/kg/day) from baseline (-23 mL/kg/day from baseline).
At 24 weeks, 38% of the patients taking the 0.05 mg/kg dose were able to reduce parenteral support infusion by at least 1 day per week. Patients also reduced their infusion time by 3 hours per day on average compared with baseline. During the study, three children (12%) who received teduglutide 0.05 mg/kg/day completely weaned off parenteral support.
The drug has a 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.
Colorectal polyps were identified during clinical trials and there is a risk for acceleration of neoplastic growth with teduglutide, the label warns. In children and adolescents, fecal occult blood testing prior to initiating treatment and annually is advised.
Colonoscopy/sigmoidoscopy is required if there is unexplained blood in the stool. Colonoscopy/sigmoidoscopy is recommended for all children and adolescents after 1 year of treatment, every 5 years thereafter while on continuous treatment with teduglutide, and if the child has new or unexplained gastrointestinal bleeding. In case of intestinal malignancy (GI tract, hepatobiliary, pancreatic), the drug should be discontinued.
Intestinal obstruction, cholecystitis, cholangitis, cholelithiasis, pancreatitis, fluid overload, and congestive heart failure have also been reported in clinical trials and postmarketing. Complete prescribing information is available online.
Cite this: FDA OKs Teduglutide (Gattex) for Children With Short Bowel Syndrome - Medscape - May 17, 2019.