FDA Approves Teduglutide for Pediatric Use

Teduglutide (Gattex) has been approved as an injection for pediatric patients 1 year of age and older with Short Bowel Syndrome.

The US Food and Drug Administration (FDA) has approved teduglutide (Gattex) as an injection for pediatric patients 1 year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous feeding.

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.

The safety profile was similar to that in adults with Short Bowel Syndrome.

"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.