In a press release on June 7, 2022, the manufacturers Regeneron Pharmaceuticals, Inc., and Sanofi announced that the FDA approved Dupixent (dupilumab) for pediatric patients aged 6 months to 5 years with moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. This approval marks the first biologic approved to treat AD in individuals from infancy to adulthood. Regulatory filings for this age group are underway by the European Medicines Agency and regulatory authorities in additional countries.
The FDA evaluated dupilumab under Priority Review, and the approval was based on phase III data. The phase III randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of dupilumab added to standard-of-care low-potency topical corticosteroid (TCS) compared with low-potency TCS alone (placebo) in 162 pediatric patients aged 6 months to 5 years with uncontrolled moderate-to-severe AD.
The primary endpoints evaluated the percentage of patients achieving an Investigator's Global Assessment score of 0 (clear) or 1 (almost clear) and 75% improvement in Eczema Area and Severity Index (also known as EASI-75) at Week 16. Additional outcome measures included itch reduction, which was measured utilizing a caregiver-reported 0 to 10 Numerical Rating Scale, with a clinically meaningful improvement defined as >4-point improvement at Week 16.
Results revealed 200 mg or 300 mg of dupilumab, based on body weight, plus low-potency TCS resulted in clear or almost clear skin in 28% of patients compared with 4% of those treated with placebo. Disease severity improved by at least 75% in 53% of patients, compared with 11% of placebo-treated patients. A clinically meaningful reduction in itch was achieved in 48% of those treated with dupilumab compared with 9% of those treated with placebo.
The safety profile of dupilumab observed through 16 weeks in children aged 6 months to 5 years was comparable to the safety profile in patients aged 6 years and older with AD. The long-term safety profile of dupilumab in children aged 6 months to 5 years through 52 weeks was also comparable with the safety profile observed in the pivotal trial and consistent with what was observed in older patients with AD. Hand-foot-and-mouth disease and skin papilloma were, respectively, reported in 5% and 2% of dupilumab patients aged 6 months to 5 years, and none of these cases led to treatment discontinuation.
Julie Block, president and CEO, National Eczema Association, stated, "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. 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."
George D. Yancopoulos, MD, PhD, president and chief scientific officer at Regeneron, and a principal inventor of Dupixent, noted, "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. Dupixent has changed the atopic dermatitis treatment paradigmÑsignificantly 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 U.S. patient aged six months or older living with this debilitating disease."
"Until today, treatment options in the U.S. for infants and children under the age of 6 suffering from moderate-to-severe atopic dermatitis have been limited to topical steroids—which may be associated with significant safety risks when used long-term. This has left patients and their caregivers in desperate need of medicines that can better address the chronic, long-term nature of the disease," Naimish Patel, MD, senior VP and head of Global Development, Immunology and Inflammation at Sanofi, stated, adding, "These young people, and their families, often struggle to cope with the significant impact itch can have not only on the body, but on many other facets of daily life. This approval means that Dupixent, with its well-established safety and efficacy profile, is now available to some of the youngest people living with this disease."
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