FDA Approves Dupilumab for Children 6 Months to 5 Years of Age With Atopic Dermatitis
Approval in this patient population makes dupilumab the first and only biologic medicine approved for the treatment of moderate-to-severe atopic dermatitis from infancy through adulthood.
The FDA has approved dupilumab (Dupixent; Regeneron Pharmaceuticals, Inc) for the treatment of children aged 6 months to 5 years with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or for when those therapies are not advisable.
The approval makes dupilumab the first and only biologic medicine FDA-approved for the treatment of moderate-to-severe atopic dermatitis from infancy through adulthood.
"Moderate-to-severe atopic dermatitis in babies and young children is more than just a rash – the intense itch can make them scratch uncontrollably throughout the day and night and cause their skin to crack and bleed," said Julie Block, president and chief executive officer of the National Eczema Association, in a press release. "Caregivers do their best to manage skincare routines multiple times a day, but for many, topical treatments are not enough. We're pleased to see how scientific innovation and research continues to address unmet needs for the atopic dermatitis community, and we're hopeful for the positive impact Dupixent can have for these children and their families."
Dupilumab was granted priority review status by the FDA for this indication. The drug is administered via subcutaneous injection at different sites.
A phase 3 randomized, double-blind, placebo-controlled trial analyzed dupilumab’s efficacy and safety when added to standard-of-care low-potency topical corticosteroids (TCS) every 4 weeks (200 mg or 300 mg, based on body weight) versus low-potency TCS monotherapy in 162 children aged 6 months to 5 years with uncontrolled moderate-to-severe atopic dermatitis.
The primary endpoints of the trial evaluated the proportion of patients achieving an Investigator's Global Assessment (IGA) score clear or almost clear skin and a 75% improvement in Eczema Area and Severity Index (EASI-75) at week 16. Secondary measures included itch reduction assessed using a caregiver-reported 0 to 10 Numerical Rating Scale, with a clinically meaningful improvement defined as ≥4-point improvement at week 16. Patients who completed the trials were eligible for an open-label extension trial to evaluate the safety and efficacy of long-term treatment with dupilumab in this patient population.
Dupilumab achieved the primary and all secondary endpoints of the trial. At 16 weeks, 28% of patients administered dupilumab with TCS achieved clear or almost-clear skin versus 4% with placebo, which was the primary endpoint. Further, 53% of patients achieved 75% or greater improvement in overall disease severity from baseline versus 11% in the control group, and 48% achieved clinically meaningful reduction in itch versus 9% in the control group.
The safety of dupilumab through 16 weeks in this patient population was similar to the profile observed in patients 6 years of age and older. Additionally, the long-term safety profile through 52 weeks was consistent with what was observed in older patients with atopic dermatitis.
Hand-foot-and-mouth disease and skin papilloma were reported in 5% and 2% of patients aged 6 months to 5 years, respectively, in the treatment group, none of which caused treatment discontinuation.
"Young children with moderate-to-severe atopic dermatitis are a significantly underserved population of patients, who spend vulnerable years of their lives suffering through the relentless and far-reaching effects of this chronic disease," said George D. Yancopoulos, MD, PhD, president and chief scientific officer at Regeneron, in a press release. "Dupixent has changed the atopic dermatitis treatment paradigm—significantly clearing skin and reducing itch—by targeting an underlying cause of this disease without broadly suppressing the immune system. Today's approval brings the proven efficacy and, importantly, well-established safety profile of Dupixent to these young children, making it the first of its kind to be approved for any US patient aged 6 months or older living with this debilitating disease."
FDA Approves Dupixent® (Dupilumab) As First Biologic Medicine For Children Aged 6 Months To 5 Years With Moderate-To-Severe Atopic Dermatitis. Regeneron. News release. June 7, 2022. https://investor.regeneron.com/news-releases/news-release-details/fda-approves-dupixentr-dupilumab-first-biologic-medicine-0