FDA approves once-weekly exenatide for children with type 2 diabetes
The FDA approved the GLP-1 receptor agonist exenatide for children aged 10 to 17 years with type 2 diabetes, the first once-weekly injectable approved for pediatric use in the United States, according to an industry press release.
The approval of exenatide extended release (Bydureon BCise, AstraZeneca) comes after positive phase 3 data, presented at the American Diabetes Association Scientific Sessions and reported by Healio, demonstrated adolescents with type 2 diabetes were more likely to achieve HbA1c targets after 24 weeks of once-weekly exenatide compared with placebo. Researchers also noted trends observed toward decreased fasting plasma glucose and reduced body weight.
“The U.S. FDA approval is an important milestone for the treatment of children with type 2 diabetes,” William Tamborlane, MD, professor and chief of pediatric endocrinology at Yale School of Medicine in New Haven, Connecticut, and the international coordinating investigator of the exenatide trial, said in the release. “Bydureon BCise brings an important new therapeutic option to physicians caring for children with this chronic disease that can lead to serious long-term issues if not adequately treated.”
For the phase 3 study, 83 adolescents aged at least 10 years with type 2 diabetes, with or without insulin or sulfonylurea therapy, were randomly assigned 5:2 once-weekly exenatide 2 mg (n = 59) or placebo (n = 24) for 24 weeks, followed by a 28-week, open-label extension phase. Primary efficacy endpoint was change from baseline HbA1c at week 24; secondary efficacy endpoints were changes in fasting glucose, body weight and systolic blood pressure. Researchers also assessed the frequency of adverse events.
At 24 weeks, once-weekly exenatide was superior to placebo in lowering HbA1c (least squares mean change, 0.36% vs. 0.49%, respectively), with a between-group difference of 0.85 percentage points (P = .012).
Researchers also noted nonsignificant least squares mean differences from baseline to 24 weeks favoring exenatide for fasting glucose (21.6 mg/dL; 95% CI, 49 to 5.7), systolic BP (2.8 mm Hg; 95% CI, 8 to 2.4) and body weight (1.22 kg; 95% CI, 3.59 to 1.15).
There were low rates of hypoglycemia during the trial, despite insulin use, and good gastrointestinal tolerability, even in the absence of exenatide titration when stating treatment. The trial was the first for a once-weekly GLP-1 receptor agonist in a pediatric population with type 2 diabetes.
“This decision is an important milestone for the care of this younger patient population by providing a convenient, once-weekly treatment option,” Mene Pangalos, executive vice president, AstraZeneca BioPharmaceuticals R&D, said in the release. “The phase 3 data that supported this approval demonstrated the safety and tolerability of exenatide extended-release in younger patients was similar to the proven safety profile of this medicine in adults.”
Exenatide extended-release was approved in the U.S. in October 2017 as a once-weekly, single-dose autoinjector device for adults with type 2 diabetes whose glucose level remains uncontrolled with one or more oral diabetes medicines in addition to diet and exercise. It was also approved for use in the European Union in August 2018.