First once-weekly GLP-1 treatment option for paediatric patients with type-2 diabetes in the EU.1
AstraZeneca’s Bydureon BCise (exenatide 2 mg prolonged-release suspension for injection in pre-filled pen) has been approved for an indication extension of its marketing authorisation in the European Union (EU), to include the treatment of type-2 diabetes (T2D) in children and adolescents 10 years and above. Bydureon BCise is now indicated in adults, adolescents and children aged 10 years and above with T2D to improve glycaemic control in combination with other glucose-lowering medicinal products including basal insulin, when the therapy in use, together with diet and exercise, does not provide adequate glycaemic control.
The approval by the European Commission (EC) is based on the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) on results from the BCB114 Phase III trial; showing in addition to standard of care, exenatide prolonged-release significantly improved glycaemic control compared to placebo.
The approval of Bydureon BCise in the EU as a once-weekly treatment option is an important development in diabetes care for paediatric patients, as the only non-insulin approved options are metformin tablets and liraglutide daily injections. With the approval of Bydureon BCise by the EC, paediatric patients and their caregivers have a convenient, once-weekly injectable suspension as an alternative.
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca said: “Paediatric type-2 diabetes patients face unique challenges in disease impact and management. As more young patients in the EU are diagnosed with type-2 diabetes every year, today’s decision is a huge step towards meeting the urgent need for effective treatment options to treat paediatric type-2 diabetes and minimise its related complications.”
The International Coordinating Investigator of the trial, William Tamborlane, MD, Department of Paediatrics, Yale School of Medicine, said: “The reported incidence of type-2 diabetes in children and adolescents is increasing worldwide, and paediatric patients managing type-2 diabetes face a higher risk of morbidity and premature mortality. Bydureon BCise offers an important new once-weekly therapeutic option to physicians caring for children with this chronic disease.”
This decision follows approval by the U.S. Food and Drug Administration (FDA) in 2021 for treatment of paediatric patients. Bydureon BCise was first approved in the EU in August 2018 as a new formulation within the marketing authorisation for Bydureon for the treatment of patients with T2D. The formulation of once-weekly Bydureon BCise is a single-dose, pre-filled pen device that is approved for use in combination with other glucose-lowering medicines, including basal insulin, to help improve glycaemic control in adults, adolescents and children aged 10 years and above with T2D whose blood sugar levels are inadequately controlled by other glucose-lowering medicines together with diet and exercise.
The BCB114 Phase III trial is the first completed trial of a once-weekly glucagon-like peptide-1 receptor agonist (GLP-1 RA) in a paediatric population with T2D. BCB114 was a 24-week, randomised, double-blind, placebo-controlled Phase III trial with a 28-week open-label extension when all placebo patients were receiving exenatide. The primary efficacy endpoint of the Phase III trial was change in glycated haemoglobin A1c (HbA1c) from baseline to week 24. Results demonstrated that patients administered exenatide prolonged-release achieved a significantly greater LS mean (SE) adjusted change in HbA1c from baseline compared to placebo (n=58, baseline A1C -0.36, n=24, baseline A1C 0.49, respectively and a with a LS mean (SE) difference of -0.85). Overall, the adverse events observed in this paediatric study were consistent with that observed in the adult population.
Notes
Type-2 diabetes
T2D is a chronic disease characterised by pathophysiologic defects leading to elevated glucose levels, or hyperglycaemia. Over time, this sustained hyperglycaemia contributes to progression of the disease. The prevalence of diabetes is projected to reach 578 million people worldwide by 2030, and 700 million by 2045. T2D accounts for approximately 90–95 percent of all cases of diagnosed diabetes.
The incidence of T2D in children and adolescents is increasing worldwide possibly due to the obesity epidemic.
BCB114 Phase III trial
BCB114 was a Phase III, double-blind, placebo-controlled, randomised, multi-centre, parallel trial assessing the safety and efficacy of exenatide prolonged-release compared to placebo in youth with T2D between 10 and <18 years of age, which showed on top of standard of care exenatide prolonged-release significantly improved glycaemic control compared to placebo in paediatrics. It is the first completed trial of a once-weekly GLP-1 RA in paediatrics with T2D. The trial included 82 children or adolescents treated with diet and exercise alone or in combination with an oral antidiabetic agent (metformin and/or sulfonylurea [SU]) and/or insulin. As clinical studies have demonstrated that once-weekly exenatide prolonged-release improved glycaemic control in adults with T2D, the BCB114 trial was designed to evaluate the effects of exenatide extended-release on glycaemic control in paediatrics with T2D.
Bydureon BCise
Bydureon BCise is an injectable prescription medicine that improves blood sugar (glucose) control in adults with T2D mellitus and should be used along with diet and exercise. Bydureon BCise is currently available in 42 countries worldwide, including European Union countries and the United States. In June 2011, Bydureon was approved for use in the EU in adults on top of other glucose-lowering treatments when these have not provided adequate control and in August 2012 was approved in the US as the first and only once-weekly treatment for T2D in adults. In August 2018, Bydureon BCise was approved in the EU as a once weekly treatment for T2D in adults.
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References
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Veeva ID: Z4-44249
Date of preparation: June 2022