Nashville, TN—Children aged 6 to 11 years had reduced rates of severe asthma attacks, improved lung function, and better control with a treatment recently approved by the FDA for that age group.

The FDA approved dupilumab as an add-on maintenance treatment for those children who have moderate-to-severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid–dependent asthma.

Dupilumab, marketed as Dupixent, is the only biologic medicine to improve lung function in children aged 6 to 11 years in a randomized phase lll trial, supporting potential as a best-in-class option. It also is the only biologic medicine approved for children with oral corticosteroid–dependent asthma.

Results of the international multicenter Liberty Asthma VOYAGE trial, published in the New England Journal of Medicine, supported FDA approval of dupilumab for the new cohort.

"This is a really important advance for children with moderate-to-severe asthma and their families," said lead investigator Leonard Bacharier, MD, an asthma specialist at Monroe Carell Jr. Children's Hospital at Vanderbilt University.

The study points out that children with moderate-to-severe asthma continue to have disease complications, even with current standard-of-care therapy. It adds that the monoclonal antibody dupilumab has been approved for the treatment of adults and adolescents with asthma as well as with other type 2 inflammatory diseases.

For the 52-week phase lll, randomized, double-blind, placebo-controlled trial, the study team assigned 408 children between the ages of 6 and 11 years who had uncontrolled moderate-to-severe asthma to receive a subcutaneous injection of dupilumab or matched placebo every 2 weeks. A dose of 100 mg was used for those weighing <30 kg and 200 mg for those weighing >30 kg. All the children also received a stable dose of standard background therapy.

Defined as the primary end point was the annualized rate of severe asthma exacerbations, while secondary end points were (1) change from baseline in the percentage of predicted prebronchodilator forced expiratory volume in 1 second (ppFEV1) at Week 12 and (2) the variation of the score on the Asthma Control Questionnaire 7 Interviewer-Administered (ACQ-7-IA) at Week 24.

End points were evaluated in the two primary efficacy populations who had either:

• A type 2 inflammatory asthma phenotype (≥150 blood eosinophils per cubic mm)
• A fraction of exhaled nitric oxide of ≥20 ppb at baseline) or
• Blood eosinophil count of at least 300 cells per cubic millimeter at baseline.

The study revealed that in patients with the type 2 inflammatory phenotype, the annualized rate of severe asthma exacerbations was 0.31 (95% confidence interval [CI], 0.22 to 0.42) with dupilumab and 0.75 (95% CI, 0.54 to 1.03) with placebo (relative risk reduction in the dupilumab group, 59.3%; 95% CI, 39.5 to 72.6; P <0.001). Researchers report that the mean (±SE) change from baseline in the ppFEV1 was 10.5±1.0 percentage points with dupilumab and 5.3±1.4 percentage points with placebo (mean difference, 5.2 percentage points; 95% CI, 2.1 to 8.3; P <0.001). They add that dupilumab also resulted in significantly better asthma control than placebo (P <0.001). "Similar results were observed in the patients with an eosinophil count of at least 300 cells per cubic millimeter at baseline. The incidence of serious adverse events was similar in the two groups," the authors advise.

"Among children with uncontrolled moderate-to-severe asthma, those who received add-on dupilumab had fewer asthma exacerbations and better lung function and asthma control than those who received placebo," according to the study funded by Sanofi and Regeneron Pharmaceuticals.

"As asthma gets increasingly severe, the burden becomes substantial, impacting the child and the entire family," Dr. Bacharier said. "While we have very good asthma therapies available, none of them are perfect in eliminating severe exacerbations."

He adds, "This is the first study of its kind in children ages 6 to 11 that has demonstrated that a biologic improves asthma exacerbations, lung function and asthma control. We were not surprised, because dupilumab was very effective in clinical trials in adults and adolescents, but we were delighted with the results and the hope they bring to children and their families."

Dr. Bacharier reveals that dupilumab was not effective for the small number of children in the trial who did not have evidence of type 2 inflammation, adding that was consistent with expectations.

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