Positive Dupixent Phase 3 Data in Children Aged 6 Months to 5 Years with Moderate-to-severe AD

Dupixent significantly improved skin clearance, and reduced itch and overall disease severity in children as young as 6 months old.

Dupixent significantly improved skin clearance, and reduced itch and overall disease severity in children as young as 6 months old, according to a new Phase 3 trial in the Lancet.

These data were the basis for the U.S. Food and Drug Administration (FDA) approval of Dupixent in June 2022 and for a regulatory submission currently under review by the European Medicines Agency.

"The Lancet's publication of these Phase 3 results is a testament to the significance of the data showing dupilumab can alleviate the multidimensional burden that moderate-to-severe atopic dermatitis places on infants, toddlers and their families," says  Amy S. Paller, M.D., Walter J. Hamlin Professor and Chair of Dermatology and Professor of Pediatrics at Northwestern University Feinberg School of Medicine, and principal investigator of the trial, in a news release. "By addressing the key inflammatory pathway driving atopic dermatitis, the trial demonstrated that dupilumab not only addressed debilitating symptoms like persistent itch and skin lesions, but also meaningfully improved sleep and reduced pain – two aspects of daily life that are critical for any child's development and well-being."

Data from this trial showed that adding Dupixent to low-potency topical corticosteroids (TCS) significantly improved skin clearance and reduced overall disease severity and itch compared to TCS alone (placebo) at 16 weeks. Additionally, Dupixent patients experienced significant improvement in measures of sleep quality and skin pain, as well as patient- or caregiver-reported outcomes and health-related quality of life. A substantially lower proportion of Dupixent patients needed rescue medications, compared to those on placebo.

Safety results through 16 weeks were similar to the safety profile in patients 6 years and older with atopic dermatitis. Adverse events that were more commonly observed with Dupixent (≥5%) included conjunctivitis (5% Dupixent, 0% placebo), herpes viral infections (6% Dupixent, 5% placebo), molluscum contagiosum (5% Dupixent, 3% placebo), rhinorrhea (5% Dupixent, 1% placebo) and dental caries (5% Dupixent, 0% placebo).  

The safety and efficacy of Dupixent in children 6 months to 5 years of age with uncontrolled atopic dermatitis has not been fully evaluated by any regulatory authority outside the U.S.

The Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent added to standard-of-care low-potency TCS compared to low-potency TCS alone in 162 children aged 6 months to 5 years with uncontrolled moderate-to-severe atopic dermatitis. Patients treated with Dupixent received either 200 mg or 300 mg (based on weight) every four weeks. 

The primary endpoints assessed the proportion of patients achieving an Investigator's Global Assessment (IGA) score of 0 (clear) or 1 (almost clear) and at least a 75% improvement in Eczema Area and Severity Index (EASI-75) at week 16.

Secondary endpoints further assessed disease measures and quality of life. Disease measures included additional EASI outcomes, itch reduction, percent of body surface area affected, skin pain, disease severity as measured by the Patient Oriented Eczema Measure, as well as SCORing Atopic Dermatitis measuring a combined assessment of disease area and severity, itch and sleep. Quality of life measures were assessed for children (by Children's Dermatology Life Quality Index for children aged 4 to 17 years and Infants' Dermatitis Quality of Life Index for children less than 4 years of age) and families (by the Dermatitis Family Impact questionnaire), as well as sleep quality.

Children who completed the trial were eligible to enroll in an open-label extension to assess the safety and efficacy of long-term treatment with Dupixent in this age group.