The Food and Drug Administration (FDA) has expanded the approval of Dupixent® (dupilumab) to include add-on maintenance treatment of patients 6 to 11 years of age with moderate to severe asthma characterized by an eosinophilic phenotype or with oral corticosteroid dependent asthma. Previously, the treatment was approved only for patients 12 years of age and older.
The approval was based on data from the randomized, double-blind, placebo-controlled phase 3 VOYAGE trial (ClinicalTrials.gov Identifier: NCT02948959), which assessed the efficacy and safety of dupilumab, an interleukin-4 receptor alpha antagonist, in 408 pediatric patients aged 6 to 11 years with uncontrolled moderate to severe asthma on a medium or high dose inhaled corticosteroid and a second controller medication or high dose inhaled corticosteroid alone.
Patients were randomly assigned 2:1 to receive either placebo or dupilumab subcutaneously 100mg every 2 weeks (less than 30kg) or 200mg every 2 weeks (30kg or greater), in addition to standard of care maintenance.
The primary endpoint was the annualized rate of severe asthma exacerbation events during the 52-week placebo-controlled period. The key secondary endpoint was the change from baseline in prebronchodilator forced expiratory volume in 1 second (FEV1) percent predicted at week 12.
Among the 259 patients who had elevated levels of eosinophils (300 cells/μL or greater), findings showed dupilumab significantly reduced the annualized rate of severe asthma exacerbation events by 65% compared with placebo (0.24 events/year vs 0.67 events/year, respectively; rate ratio, 0.35 [95% CI, 0.22-0.56]). In patient with baseline blood eosinophil count less than 150 cells/μL and FeNO less than 20 ppb, similar severe asthma exacerbation rates were observed between dupilumab and placebo.
In the dupilumab treatment arm, significant improvements in percent predicted prebronchodilator FEV1 were observed as early as 2 weeks and were maintained through week 52. A clinically meaningful improvement in asthma control was also reported in 81% of patients treated with dupilumab at week 24 compared with 64% of the placebo group, as measured by at least a 0.5 improvement on a 7-point scale.
The most common adverse reactions with dupilumab were injection site reactions, viral upper respiratory tract infections, and eosinophilia.
“Despite available treatments, moderate to severe asthma can severely impact children’s developing airways, causing sleepless nights, persistent coughing and wheezing, and potentially life-threatening exacerbations that require the use of systemic steroids that can negatively affect growth,” said George D. Yancopoulos, MD, PhD, President and Chief Scientific Officer at Regeneron. “This approval means that Dupixent, a first-of-its-kind treatment with a well-established efficacy and safety profile, can now be used by younger children with certain types of moderate to severe asthma in the US.”
- FDA expands approval of Dupixent® (dupilumab) to include children aged 6 to 11 years with moderate-to-severe asthma. News release. Regeneron Pharmaceuticals, Inc. October 20, 2021. Accessed October 21, 2021. https://www.prnewswire.com/news-releases/fda-expands-approval-of-dupixent-dupilumab-to-include-children-aged-6-to-11-years-with-moderate-to-severe-asthma-301405141.html.
- Dupixent. Package insert. Sanofi-aventis US LLC and Regeneron Pharmaceuticals, Inc.; 2021. Accessed October 21, 2021. https://www.regeneron.com/downloads/dupixent_fpi.pdf.