FDA Expands Dupilumab to Children Aged 2 to 11 Years With Uncontrolled CSU

The US Food and Drug Administration (FDA) has approved dupilumab (Dupixent) for children aged 2 to 11 years with chronic spontaneous urticaria (CSU) that remains symptomatic despite H1 antihistamine treatment, according to a joint announcement from Regeneron and Sanofi.1

The decision, which was announced on April 22, 2026, extends the drug’s prior US CSU indication in adults and adolescents aged 12 years and older.

“With this approval, Dupixent has become the first biologic medicine in the U.S. for young children suffering from uncontrolled chronic spontaneous urticaria, an unpredictable skin disease that impacts quality of life during these children’s most formative years,” George D. Yancopoulos, MD, PhD board co-chair, president, and chief scientific officer at Regeneron, and a principal inventor of Dupixent, said in a statement.1

Dupilumab Trials in Pediatric CSU

According to the announcement, the pediatric label expansion was based primarily on the phase 3 LIBERTY-CUPID program, using efficacy and safety extrapolation from randomized trials in older patients and pharmacokinetic data from the single-arm CUPIDKids study in children aged 2 to 11 years.1

In Study A and Study C, both replicate double-blind, placebo-controlled trials in patients aged 6 years and older who were symptomatic despite antihistamines and naïve to anti-IgE therapy, dupilumab significantly improved weekly itch severity score (ISS7) and weekly urticaria activity score (UAS7) vs placebo at week 24, the companies said. In adults and adolescents, dupilumab also increased the likelihood of well-controlled disease (UAS7 ≤6) and complete response (UAS7 = 0) at 24 weeks.1

Study B enrolled patients aged 12 years and older who were symptomatic despite antihistamines and were inadequate responders or intolerant to anti-IgE therapy, providing additional safety data, according to the release. CUPIDKids was a single-arm phase 3 trial designed to assess safety, efficacy, and pharmacokinetics over 24 weeks; its primary endpoint was serum dupilumab concentration over time, including trough levels at weeks 12 and 24. No numerical efficacy results from CUPIDKids were disclosed in the release.1

Additional Considerations for Dupilumab in CSU

International guidance generally recommends second-generation H1 antihistamines as first-line therapy, with dose escalation and then biologic treatment for refractory disease. Omalizumab has been a standard biologic option for antihistamine-refractory CSU in older patients, although pediatric options for children younger than 12 years have been more constrained.

Dupilumab is a fully human monoclonal antibody that blocks interleukin 4 and interleukin 13 signaling. It is already approved across multiple type 2 inflammatory diseases, including atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, and eosinophilic esophagitis.1 Its role in CSU has been of interest because not all patients respond adequately to antihistamines or anti-IgE therapy, and CSU pathobiology may include overlapping inflammatory pathways beyond mast cell and IgE-mediated signaling.

The sponsors described safety findings in the CSU development program as generally consistent with the established dermatologic safety profile of dupilumab.1 In Studies A, B, and C, the most common adverse event (AE) occurring more often with dupilumab than placebo was injection-site reaction.1 The companies also said no new AEs were identified in children aged 2 to 11 years with CSU, with pediatric safety additionally supported by experience in other approved indications.1

“Dupixent is now approved for 9 different allergy-related conditions, from asthma to atopic dermatitis, and this is the fifth of these indications now extended to young children,” The FDA’s authorization reinforces our medicine’s well-established safety profile and potential to transform outcomes for chronic diseases driven in part by type 2 inflammation impacting some of the most vulnerable populations. As the most widely used innovative branded antibody medicine, Dupixent has the potential to change yet another treatment paradigm.”

References

1. Regeneron Pharmaceuticals, Inc; Sanofi. Dupixent (dupilumab) approved in the U.S. as the first biologic medicine for young children with uncontrolled chronic spontaneous urticaria (CSU). GlobeNewswire. April 22, 2026. Accessed April 22, 2026. https://www.globenewswire.com/news-release/2026/04/22/3279470/0/en/dupixent-dupilumab-approved-in-the-u-s-as-the-first-biologic-medicine-for-young-children-with-uncontrolled-chronic-spontaneous-urticaria-csu.html

2. Wechsler ME, Ford LB, Maspero JF, et al. Long-term safety and efficacy of dupilumab in patients with moderate-to-severe asthma: LIBERTY ASTHMA TRAVERSE. Eur Respir J. 2022;60(3):2102791.