The US FDA approved Dupixent (dupilumab) for the treatment of chronic spontaneous urticaria in children aged two to 11 years, marking the first biologic therapy approved for this age group in the indication. The approval, granted to Sanofi and Regeneron Pharmaceuticals, extends an existing authorization that covered adults and adolescents aged 12 years and older, and represents the fifth type 2 inflammation-driven disease for which dupilumab has received approval in children under 12.
The authorization covers children who remain symptomatic despite treatment with H1 antihistamines (H1AH), the current standard first-line therapy for CSU. Dosing is weight- and age-based: children aged two to five years receive 200 mg every four weeks if weighing between 5 kg and less than 15 kg, or 300 mg every four weeks if weighing 15 kg to less than 30 kg, without an initial loading dose. Children aged six to 11 years follow a separate weight-tiered schedule, with doses ranging from 300 mg Q4W to 300 mg Q2W following an initial loading dose. The drug is administered subcutaneously and can be given at home by a caregiver after appropriate training.
The approval was based primarily on data from the LIBERTY-CUPID Phase III clinical study program, which included four studies. Study A and Study C were replicate, double-blind, placebo-controlled trials assessing dupilumab as add-on therapy to antihistamines in patients aged six years and older who were antihistamine-naïve to anti-IgE therapy and remained symptomatic. Study B evaluated patients aged 12 and older who were inadequate responders or intolerant to anti-IgE therapy. The CUPIDKids study was a single-arm Phase III trial in children aged two to 11 years, with a primary endpoint measuring serum dupilumab concentration over time, including trough levels at weeks 12 and 24. Efficacy in the pediatric age group was established through extrapolation from Study A and Study C, supported by the pharmacokinetic data from CUPIDKids. In Study A and Study C, dupilumab significantly reduced itch severity and urticaria activity, as measured by the weekly urticaria activity score (UAS7), compared to placebo at week 24, and increased the proportion of patients achieving well-controlled disease status or complete response. Safety in the two-to-11 age group was further supported by pediatric data from other approved dupilumab indications. The most common adverse reaction reported at a rate of 2% or greater, and more frequently than placebo, was injection site reactions. No new adverse reactions were identified in the pediatric CSU population.
The approval addresses a treatment gap that has persisted in pediatric CSU management. More than 14,000 children aged two to 11 in the US are estimated to have CSU that remains uncontrolled despite antihistamine use, the companies said. Prior to this authorization, the only approved biologic for CSU — omalizumab (Xolair), an anti-IgE monoclonal antibody — was not indicated for children under 12 in this condition, leaving younger patients without a licensed biologic option.
The CSU indication now joins atopic dermatitis, asthma, eosinophilic esophagitis, and prurigo nodularis as type 2 inflammation-driven conditions for which dupilumab carries pediatric approval below age 12. The drug has also received approval in the EU and other markets for CSU in children aged two to 11. Sanofi and Regeneron report that more than 1.4 million patients are currently receiving dupilumab globally across all approved indications, and the development program spans more than 60 clinical studies involving more than 12,000 patients. Active Phase III investigations continue in chronic pruritus of unknown origin and lichen simplex chronicus, indications where regulatory authorization has not yet been sought.