FDA Approves Dupilumab for Chronic Spontaneous Urticaria in Children Aged 2 to 11

The US FDA has approved dupilumab (Dupixent; Regeneron/Sanofi) for children aged 2 to 11 years with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine (H1AH) therapy — making it the first biologic authorized in the US for this age group.^1,2

The decision extends a prior approval that covered adults and adolescents 12 and older. For the more than 14,000 US children in this age range estimated to have antihistamine-refractory CSU, it fills a treatment gap that has long left clinicians with few options beyond cycling through symptom-focused therapies.³

Why This Matters Mechanistically

CSU is a chronic inflammatory skin disease driven in part by type 2 inflammation, the same pathway implicated in atopic dermatitis and asthma. It presents as sudden, recurring hives and itch that can be deeply disruptive, particularly in young children.

Standard H1AHs manage symptoms but don't address the underlying inflammatory drivers. Dupilumab works upstream, blocking IL-4 and IL-13 signaling — 2 cytokines central to type 2 inflammation. That mechanistic distinction is part of what makes this approval meaningful: it's not another antihistamine-adjacent option, it's a different approach entirely.

How the Approval Was Built

The FDA's decision is grounded in data from the LIBERTY-CUPID phase 3 program, which includes 4 studies: CUPIDKids (NCT05526521), Study A, Study B, and Study C.^1,2

The core efficacy case came from Study A and Study C — replicate, double-blind, placebo-controlled trials in patients aged 6 and older who were antihistamine-refractory and anti-IgE naïve. Both trials evaluated dupilumab as add-on therapy to standard-of-care antihistamines over 24 weeks. Dosing was 300 mg every 2 weeks following an initial loading dose, or 200 mg every 2 weeks for pediatric patients weighing 30 kg to less than 60 kg.

At week 24, dupilumab significantly reduced itch severity — the primary endpoint, measured by the weekly Itch Severity Score (ISS7, 0–21) — and urticaria activity via the UAS7 (0–42 scale) as the key secondary endpoint. Patients on dupilumab were also more likely to achieve well-controlled disease status (UAS7 ≤6) or complete response (UAS7 = 0) compared to placebo. Study B contributed additional safety data in patients 12 and older who were inadequate responders to, or intolerant of, anti-IgE therapy.

The Pediatric Data

For the 2-to-11 age group specifically, the single-arm CUPIDKids trial assessed pharmacokinetics, safety, and efficacy in children with antihistamine-refractory CSU. Dosing was age- and weight-based — 200 mg every 2 or 4 weeks, or 300 mg every 4 weeks, with or without a loading dose. The trial's primary endpoint was pharmacokinetic, measuring serum dupilumab concentrations including Ctrough at weeks 12 and 24. Efficacy was then extrapolated from the adult and adolescent data, a regulatory approach that's become more common in pediatric drug development. Safety for this age group was further supported by pediatric data across dupilumab's other approved indications.

Safety Profile

Across all 4 LIBERTY-CUPID trials, the safety findings were consistent with dupilumab's established profile in its approved dermatologic indications. Injection site reactions were the most commonly reported adverse event (≥2%) occurring at a higher rate than placebo in Studies A, B, and C. No new safety signals were identified in children aged 2 to 11.

The Bigger Picture

This marks dupilumab's 9th approved indication in the US and the 5th extended to children under 12. The CSU approval in this pediatric age group is also in effect in the EU and select other countries.

For dermatology clinicians managing young patients who've exhausted antihistamine options, this approval introduces a biologic backed by a well-characterized safety record, and a mechanism that actually targets the disease rather than just its symptoms.

References

1. Sanofi and Regeneron’s Dupixent approved in the US as the first biologic medicine for young children with uncontrolled chronic spontaneous urticaria. News release. Sanofi. Published April 22, 2026. Accessed April 22, 2026. https://www.news.sanofi.us/2026-04-22-Sanofi-and-Regenerons-Dupixent-approved-in-the-US-as-the-first-biologic-medicine-for-young-children-with-uncontrolled-chronic-spontaneous-urticaria
2. Dupixent® (dupilumab) approved in the US as the first biologic medicine for young children with uncontrolled chronic spontaneous urticaria (CSU). News release. Regeneron. Published April 22, 2026. Accessed April 22, 2026. https://investor.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-approved-us-first-biologic-medicine-young
3. Ensina LF, Brandão LS, Melo ACDB, Antila M, Ben-Shoshan M, Solé D. Chronic urticaria treatment challenges in children. Rev Paul Pediatr. 2024;43:e2024107. Published 2024 Dec 20. doi:10.1590/1984-0462/2025/43/2024107