In a decision that expands the April 2025 approval of dupilumab (Dupixent, Regeneron/Sanofi) for chronic spontaneous urticaria (CSU) in adults and adolescents aged 12 years and older, the U.S. Food and Drug Administration (FDA) has approved dupilumab for children aged 2 to 11 years with CSU and persistent symptoms despite H1 antihistamine treatment.
“Dupixent is the first biologic approved for patients as young as 2 years of age, offering a targeted approach that inhibits [interleukin]-4 and IL-13 signaling, two key and central drivers of the type 2 inflammation that contributes to this disease,” explained Alyssa Johnsen, MD, PhD, global therapeutic area head of immunology development at Sanofi.
A Much-Needed Addition
CSU is a chronic inflammatory skin disease driven in part by type 2 inflammation that affects approximately 1.7 million people in the United States. It is characterized by unpredictable red, swollen, itchy hives that recur for six weeks or more without an identifiable cause.
CSU is typically treated with H1 antihistamines (H1AH), which target H1 receptors on cells to help control symptoms of itch and urticaria. However, the disease remains uncontrolled despite H1AH treatment in more than 14,000 children in the U.S. aged 2 to 11 years, leaving some with limited treatment options. These patients may continue to experience debilitating symptoms that significantly impact quality of life.
“Before this approval, only antihistamines were approved for children younger than 12 years of age,” noted Thomas B. Casale, MD, professor of medicine and pediatrics at University of South Florida in Tampa and an investigator for the LIBERTY-CUPID clinical trial program. “Since about 50% of patients do not adequately respond to antihistamines alone, this leads to a gap in the treatment of these children.”
Dr. Casale added that because CSU is associated with anxiety and decreased quality of life, “providing a therapeutic option for children with antihistamine unresponsiveness is a much-needed addition to our therapeutic armamentarium.”
Brian S. Kim, MD, MTR, Sol and Clara Kest Professor of Dermatology and vice chair of research at Icahn School of Medicine at Mount Sinai, as well as director of the Mark Lebwohl Center of Neuroinflammation and Sensation, also commented on the approval.
“For families of young children with CSU, the unpredictability of hives and severe itch can dominate everyday life,” he observed. “This new approval of Dupixent for kids as young as 2 years, offers, for the first time, a biologic therapy designed to address the underlying type 2 inflammation driving CSU — not just mask the symptoms.”
Details and Data
The expanded approval was primarily supported by data from the LIBERTY-CUPID clinical study program, including two phase 3 studies, Study A and Study C, that included adults and adolescents aged 12 years and older with CSU, as well as pharmacokinetic data from the phase 3 CUPIDKids study involving children aged 2 to 11 years with CSU.
For 24 weeks, participants in Study A and Study C received dupilumab 200 mg every two or four weeks, or 300 mg every four weeks, based on weight and age. At the end of Study A, dupilumab reduced itch severity by 63% and urticaria activity by 65% (P < .001). In contrast, placebo reduced itch by 35% and urticaria activity by 37%.
Additional findings from Study C showed that a higher proportion of participants receiving dupilumab achieved well-controlled disease status compared with placebo (41% vs. 23%; P = .005), as well as complete response rates (30% vs. 18%; P = .02).
Study B in the LIBERTY-CUPID program showed that dupilumab’s safety profile was consistent with its known safety profile across approved dermatologic indications. The most common adverse event was injection-site reactions, occurring in 2% or more of participants. Investigators did not observe any new safety events in children aged 2 to 11 years.
Key takeaways:
- Approval of dupilumab has been expanded to include children aged 2 to 11 years with CSU.
- The approval was supported by data from the LIBERTY-CUPID clinical trial program.
“Dupixent is now approved for nine different allergy-related conditions, from asthma to atopic dermatitis, and this is the fifth of these indications now extended to young children,” said George D. Yancopoulos, MD, PhD, board co-chair, president, and chief scientific officer at Regeneron.
The expanded approval of Dupixent may offer a promising new option for younger patients living with CSU and provide much-needed reassurance for families and caregivers managing this chronic condition.
