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FDA Accepts NDA for Expanded use of Gattex for Short Bowel Syndrome
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The FDA has accepted the filing of a new drug application to extend the use of Gattex for injection in children aged 1 year to 17 years with short bowel syndrome, according to a company press release.
Gattex (teduglutide [rDNA origin], Shire) is indicated for adults with SBS who are dependent on parenteral support.
“The filing acceptance of Shire’s supplemental new drug application by the U.S. FDA is an important milestone in our efforts to investigate the use of Gattex as a potential treatment for children with SBS who are dependent on parenteral support,” Howard Mayer, MD, Shire’s Senior Vice President and Chief Medical Officer, said in the release.
The new drug application includes data from two completed phase 3 studies as well as preliminary data from two ongoing extension studies, according to the release.
One of the completed studies included in the application was a 24-week, double-blind study of 59 children aged 1 year to 17 years who either received 0.025 mg/kg of teduglutide (n = 24), 0.05 mg/kg of teduglutide (n = 26) or standard care (n = 9) for the treatment of their SBS.
Results indicated that 54.2% of patients who received 0.025 mg/kg of teduglutide, and 69.2% of patients who received 0.05 mg/kg of teduglutide achieved a 20% or greater reduction in the volume of parenteral support at 24 weeks compared with baseline, which served as the study’s primary endpoint.
Only 11.1% of patients who received standard of care reported achieving the same reduction as those who received either dose of teduglutide.
“Many children who develop SBS as infants may remain dependent on parenteral support, which helps to address their nutrition and fluid needs,” Beth Carter, MD, medical director of Intestinal Rehabilitation and Nutrition Support at Children’s Hospital Los Angeles, said in the release. “While the goal in SBS management is to ensure patients receive the right amount of nutrients and fluids, we also look for opportunities to reduce, and even help some of these patients wean off of parenteral support. The development of teduglutide for pediatric SBS patients age[d] 1 year and older is important as the data from the phase 3 studies suggest it might potentially address an unmet medical need in this patient population.”
The FDA is expected to reach a decision on the extended indication of Gattex in March 2019, according to the release.