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.
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.
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.
Teduglutide was first approved in 2012 as a subcutaneous injection for adults with Short Bowel Syndrome.
A rare, chronic disorder, Short Bowel Syndrome is life-threatening because patients are unable to absorb sufficient nutrients and fluids from eating and drinking. Instead, they must rely on intravenous feeding, or parenteral support (PS). The condition occurs when a large portion of the intestine must be surgically removed due to congenital or acquired conditions, or due to trauma.
“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. “I’m pleased that patients have access to a medication that may help them reach that goal.”
The FDA’s decision to approve teduglutide was supported by data from a 24-week study. The study included 59 children ages 1 to 17 with Short Bowel Syndrome. Participants chose whether to receive teduglutide or standard of care treatment (n = 9). Those who chose teduglutide were randomized to 0.025 mg/kg/day (n = 24) or 0.05 mg/kg/day (n = 26).
At 24 weeks, 69% of participants (18 of 26) receiving teduglutide .05 mg/kg each day achieved at least a 20% reduction in parenteral support volume and 38% (10 of 26) reduced PS infusion by >1 day per week. Children receiving teduglutide .05 mg/kg each day also experienced a 42% mean reduction in PS volume from baseline.
During the study, 12% (3 of 26) of children in the teduglutide .05 mg/kg/day group were able to stop intravenous feeding completely.
“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. This approval underscores Takeda’s commitment to patients with rare and devastating GI conditions like SBS,” said Andrew Grimm, Global Clinical Development Lead, Takeda.
The company Press Release can be found here.