The drug has potential to be best-in-class treatment option for children aged 6-11 years with uncontrolled moderate-to-severe asthma.
The data further suggested that the fully-human monoclonal antibody has the potential to be best-in-class treatment option for this patient population.
Investigators at Sanofi and Regeneron Pharmaceuticals conducted the LIBERTY ASTHMA VOYAGE study, a Phase 3, randomized, double-blind, placebo-controlled trial, which assessed the efficacy and safety of dupilumab in children aged 6-11 years
The treatment was used in addition to standard-of-care maintenance therapy of medium-dose inhaled corticosteroid (ICS) with a second controller medication. Dupilumab was also assessed in patients who used high-dose ICS with or without a second controller medication.
Overall, the investigators enrolled a total of 408, with 259 patients having a baseline EOS ≥300 cells/µl. Furthermore, 350 patients had markers of type 2 inflammation (baseline EOS ≥150 cells/μl or FeNO ≥20 ppb).
Additionally, there was no minimum biomarker required for enrollment.
Patients were then randomized 1:1 to receive subcutaneously either dupilumab or placebo every 2 weeks during the 52-week treatment period. Those in the dupilumab cohort who were ≤30 kg received 100 mg of the injection, and those who were >30 kg received 200 mg.
Thus, over the course of 1 year, the investigators noted a 65% (P<.0001) average reduction in rate of severe asthma attacks in the population who had received 100 mg of the investigative drug. The population who had received 200 mg saw a 59% (P<.0001) average reduction.
The results showed 0.24 (100 mg) and 0.34 (200 mg) events per year occurred in the dupilumab cohort versus 0.67 and 0.75 in placebo, respectively.
And finally, for the dupilumab cohort, significant improvement in lung function was seen as early as week 2 and was sustained for up to week 52. Thus, at week 12, improvement in lung function compared to baseline increased by 10.15 and 10.53 percentage points.
As for the placebo group, the improvement was only 4.83 and 5.32 percentage points.
A total of 83% in the dupilumab cohort experienced adverse events—versus 80% with placebo. The most common events were injection site reactions (18% dupilumab, 13% placebo), viral upper respiratory tract infections (12% dupilumab, 10% placebo), and eosinophilia (6% dupilumab, 1% placebo).
"Children with uncontrolled moderate-to-severe asthma often struggle to breathe, largely because of their impaired lung function, and this can have a serious impact on a child's quality of life. It not only reduces their ability to participate in day-to-day activities, but can also take a huge emotional toll on the child and their family," said John Reed, MD, PhD, Global Head of Research and Development, Sanofi, in a statement.
"Dupixent is the only biologic shown in a controlled Phase 3 trial to improve lung function in children, which is generally consistent with results seen in the adolescent and adult trials. These positive data are especially encouraging for younger children who are struggling to manage their uncontrolled asthma."
US and EU regulatory submissions for children 6-11 years old are planned for early 2021.