FDA Approves Dupixent for Children 6 Months to 5 Years with Moderate to Severe Atopic Dermatitis

Dupixent is the first and only biologic medicine approved to treat moderate-to-severe atopic dermatitis from infancy through adulthood.

The FDA has approved Regeneron Pharmaceuticals, Inc. and Sanofi's Dupixent (dupilumab) for children aged 6 months to 5 years with moderate to severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. Regulatory filings for this age group are underway by the European Medicines Agency and regulatory authorities in additional countries.

"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," says Julie Block, President and Chief Executive Officer at National Eczema Association, in a news 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."

Atopic dermatitis is a chronic type 2 inflammatory skin disease. Eighty-five to ninety percent of patients first develop symptoms before 5 years of age, which can often continue through adulthood. Symptoms include intense, persistent itch and skin lesions that cover much of the body, resulting in skin dryness, cracking, pain, redness or darkening, and crusting and oozing. In the U.S., more than 75,000 children aged 5 years and younger have uncontrolled moderate-to-severe disease and are most in need of new treatment options. Moderate to severe atopic dermatitis may also significantly impact the quality of life of a young child and their caregivers.

"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," says George D. Yancopoulos, MD, PhD, President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. "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 U.S. patient aged six months or older living with this debilitating disease."

The FDA evaluated Dupixent under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. The approval is based on data that include a Phase 3 trial evaluating Dupixent every four weeks (200 mg or 300 mg, based on body weight) plus low-potency topical corticosteroids (TCS) or TCS alone. The trial met the primary and all secondary endpoints. At 16 weeks, patients who received Dupixent with TCS experienced the following, compared to TCS alone (placebo):

• 28% achieved clear or almost-clear skin compared to 4% with placebo, the primary endpoint.
• 53% achieved 75% or greater improvement in overall disease severity from baseline compared to 11% with placebo, the co-primary endpoint outside of the U.S.
• 48% achieved clinically meaningful reduction in itch compared to 9% with placebo.

The safety profile of Dupixent observed through 16 weeks in children aged 6 months to 5 years was similar to the safety profile in patients 6 years and older with atopic dermatitis. The long-term safety profile of Dupixent in children aged 6 months to 5 years through 52 weeks was also similar to the safety profile observed in the pivotal trial and consistent with what was observed in older patients with atopic dermatitis. Hand-foot-and-mouth disease and skin papilloma were, respectively, reported in 5% and 2% of Dupixent patients aged 6 months to 5 years, and none of these cases led to treatment discontinuation.

"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," says Naimish Patel, MD, Senior Vice President, Head of Global Development, Immunology and Inflammation at Sanofi. "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."