FDA approves extension of indication of Gattex in Short Bowel Syndrome

Takeda Pharmaceuticals announced that the FDA approved extending the indication of Gattex (teduglutide) for injection to pediatric patients 1 year of age and older with Short Bowel Syndrome (SBS) who need additional nutrition or fluids from intravenous (IV) feeding (parenteral support).

In a 24-week pediatric study, Gattex helped reduce the volume of daily PS required and time spent administering PS. Some children even achieved complete freedom from PS. Fifty- nine pediatric patients with SBS aged 1 year through 17 years chose whether to receive Gattex or standard of care (SOC). Patients who chose to receive Gattex 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. The recommended dosage of Gattex is 0.05 mg/kg/day. Randomization to the Gattex dose groups was stratified by age.

At the end of the 24-week study, 69% of patients (18/26) who took Gattex 0.05 mg/kg each day reduced PS volume by 20% or more. Based on patient-diary data, patients who received Gattex 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 3 out of 26 (12%) children who received Gattex 0.05 mg/kg/day completely weaned off PS.

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

Comment: Gattex is the first and only medicine that mimics naturally occurring glucagon-like peptide-2 (GLP-2), which helps the remaining intestine absorb more nutrients. In a pharmacodynamic study in adults, Gattex was shown to improve the amount of fluids absorbed by the intestines.