FDA extends approval of dapagliflozin to Childers for treatment of type 2 diabetes

The US food and Drug Administration has approved dapagliflozin for treatment of children and adolescents with type 2 diabetes as an add-on treatment to metformin and insulin.

The drug produced by AstraZenaca under the brand name Farxiga and previously approved for use in adults lowered the A1C significantly when compared to study participant controls in clinical trials.

The FDA approval was based on positive results from the paediatric T2NOW Phase III trial. Farxiga was previously approved in the US in adults with T2D as an adjunct to diet and exercise to improve glycaemic control.

Ruud Dobber, Executive Vice President, BioPharmaceuticals Business Unit, AstraZeneca, said: “The prevalence of type-2 diabetes continues to rise in children and adolescents, yet oral treatment options have remained limited for this population. Today’s approval represents an important milestone for paediatric patients living with type-2 diabetes in the US, extending this medicine’s potential benefits to even more patients facing high unmet needs and reinforcing AstraZeneca’s commitment to delivering innovative treatments across cardiovascular, renal and metabolic diseases.”

T2D is a chronic disease affecting people of all ages. The incidence and prevalence of T2D in children and adolescents are increasing globally. In the US, there are nearly 30,000 patients under 20 living with T2D with 5,300 new cases diagnosed each year, according to the US Centers for Disease Control and Prevention and recent research. Younger patients often experience earlier onset of complications and faster advancement of disease compared to adults with the same condition.

Data from the T2NOW Phase III trial, published in The New England Journal of Medicine Evidence, demonstrated a significant reduction in A1C, a marker of average blood sugar, for patients treated with Farxiga compared to patients receiving placebo. Adjusted mean change in A1C was -0.62% for Farxiga versus +0.41% for placebo, a difference of -1.03% (95% CI: -1.57-0.49; p<0.001).2 Statistical significance was achieved in the primary endpoint and in all secondary endpoints versus placebo at week 26, demonstrating Farxiga can provide clinically meaningful improvements in glycaemia for children and adolescents with T2D. The safety results in this patient population were consistent with those in adults with T2D, in line with the well-characterised safety profile for Farxiga.

Farxiga, a first-in-class, oral, once-daily sodium-glucose cotransporter 2 (SGLT2) inhibitor, was approved in 126 countries, including the EU (marketed under the brand name Forxiga), as an adjunct to diet and exercise to improve glycaemic control in adults with T2D.

Forxiga is also approved for paediatric patients aged 10 years and above with T2D in 56 countries, including the EU and other regions, based on results from the T2GO Phase III clinical trial.

Additional regulatory submissions and rollout plans are under consideration pending further market evaluations.

T2D

T2D is a chronic disease characterised by pathophysiologic defects leading to elevated glucose levels, or hyperglycaemia. Over time, this sustained hyperglycaemia contributes to further progression of the disease. The prevalence of diabetes is projected to reach 783 million by 2045. T2D is the most common type of diabetes, accounting for over 90% of all diabetes worldwide. Significant unmet medical need still exists, as many patients have poor blood sugar control and low medication adherence.

T2NOW

T2NOW was a randomised, double-blind, placebo-controlled Phase III trial designed to evaluate the efficacy and safety of dapagliflozin as add-on treatment in children and adolescents with T2D receiving metformin, insulin or both. Patients were randomised to dapagliflozin, saxagliptin or placebo. Those receiving an active drug were further randomised to continue their current dose, or up-titrate to a higher dose of the same active treatment. The primary endpoint was change in A1C after 26 weeks vs placebo for dapagliflozin (5 or 10 mg) or saxagliptin (2.5 or 5 mg).2 Secondary endpoints included change in fasting plasma glucose and proportion of patients (A1C ≥7% at baseline) achieving A1C <7.0% (53 mmol/mol) after 26 weeks.

Farxiga

Farxiga (dapagliflozin) in the US and marketed as Forxiga in the rest of world, is a first-in-class, oral, once-daily sodium-glucose cotransporter 2 (SGLT2) inhibitor. As of June 2024, Forxiga was approved in 126 countries as an adjunct to diet and exercise to improve glycaemic control in adults with T2D. Forxiga is approved for paediatric patients aged 10 years and above with T2D in the EU and other countries based on the T2GO trial.

In addition, Forxiga is approved for the treatment of heart failure across the full ventricular ejection fraction range (HFrEF and HFpEF) and CKD in adult patients in more than 100 countries around the world. Forxiga was the first heart failure medication to demonstrate mortality benefit across the full ejection fraction range.

Research has shown Forxiga’s efficacy in preventing and delaying cardiorenal disease, while also protecting the organs – important findings given the underlying links between the heart, kidneys and pancreas. Damage to one of these organs can cause the other organs to fail, contributing to leading causes of death worldwide, including T2D, heart failure (HF) and chronic kidney disease (CKD).