FDA approves dupilumab for chronic spontaneous urticaria in children 2 years and older

The FDA has approved dupilumab (Dupixent; Regeneron/Sanofi) for the treatment of chronic spontaneous urticaria (CSU) in children aged 2 to 11 years who remain symptomatic despite H1 antihistamine (H1AH) therapy, making it the first biologic medicine available for young children with this condition.

The approval, announced on April 22, 2026, expands the drug’s existing CSU indication for adults and adolescents 12 years and older, which was granted in April 2025.

“With this approval, Dupixent has become the first biologic medicine in the US 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,” said George D. Yancopoulos, MD, PhD, board cochair, president, and chief scientific officer at Regeneron.¹

The regulatory decision was based primarily on data from the LIBERTY-CUPID clinical trial program, which included efficacy and safety extrapolation from 2 phase 3 trials (study A and Sstudy C) conducted in adults and adolescents, complemented by pharmacokinetic data from CUPIDKids, a single-arm, phase 3 trial enrolling children aged 2 to 11 years with CSU.

In study A and study C, which were replicate, double-blind, placebo-controlled trials in patients 6 years and older who were naïve to anti-immunoglobulin E (IgE) therapy, dupilumab significantly reduced itch severity, as measured by the weekly itch severity score (ISS7; 0-21 scale), the primary end point at week 24. The key secondary end point, change from baseline in the weekly urticaria activity score (UAS7; 0-42 scale), a composite of itch and hives, also demonstrated significant improvement compared with placebo. Dupilumab additionally increased the likelihood of achieving well-controlled disease (UAS7 ≤6) or complete response (UAS7 = 0) at week 24.¹

An additional study, study B, evaluated dupilumab in patients 12 years and older who were inadequate responders or intolerant to anti-IgE therapy and who remained symptomatic despite antihistamine use. This trial contributed supplementary safety data.¹

The CUPIDKids trial assessed the safety, efficacy, and pharmacokinetics of dupilumab in children aged 2 to 11 years with CSU who remained symptomatic despite antihistamine therapy. During the 24-week treatment period, dosing was weight- and age-based, ranging from 200 mg every 2 or 4 weeks to 300 mg every 4 weeks. The primary end point measured serum drug concentrations, including trough levels at weeks 12 and 24. Safety in this pediatric population was further supported by data from children enrolled in other approved dupilumab indications.¹

Across all 4 CSU trials, safety results were generally consistent with the known safety profile of dupilumab in its approved dermatologic indications. The most common adverse reaction observed more frequently with dupilumab than placebo (≥2%) was injection site reactions. No new adverse reactions were identified in children aged 2 to 11 years.¹

CSU is a chronic inflammatory skin disease driven in part by type 2 inflammation, characterized by the sudden onset of debilitating hives and recurring itch. While H1AH therapy represents the standard first-line approach, more than 14,000 children aged 2 to 11 years in the United States continue to have uncontrolled disease despite this treatment, according to the manufacturers. Prior to this approval, these children had limited alternative treatment options.¹

The drug is now approved for 9 allergy-related conditions in the United States, and CSU represents the fifth indication extended to children younger than 12 years.¹

Alyssa Johnsen, MD, PhD, global therapeutic area head of immunology development at Sanofi, noted that the approval addresses a significant gap. “Until now, these patients had to rely on limited treatment options that didn’t address potential critical mediators of chronic spontaneous urticaria.”¹

REFERENCES

1. Regeneron Pharmaceuticals, Inc. Dupixent (dupilumab) approved in the U.S. as the first biologic medicine for young children with uncontrolled chronic spontaneous urticaria (CSU). News release. 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. 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. April 22, 2026. Accessed April 22, 2026. https://www.sanofi.com/en/media-room/press-releases/2026/2026-04-22-21-30-00-3279472