FDA Approves Dupilumab for Young Children With Uncontrolled CSU

The FDA has approved dupilumab (Dupixent; Sanofi/Regeneron) for the treatment of children aged 2 to 11 years with chronic spontaneous urticaria (CSU) who remain symptomatic despite histamine-1 antihistamine (H1AH) treatment, making it the first biologic approved for young children with this condition, according to a press release.¹

The approval extends the indication previously approved in April 2025 for patients 12 years and older with uncontrolled CSU, which was a landmark decision that represented the first new targeted therapy for the condition in over a decade.²

CSU is a chronic inflammatory skin disease that drives sudden, unpredictable hives and recurring itch, in part due to type 2 inflammation.¹ More than 14,000 children in the US between the ages of 2 and 11 have uncontrolled CSU despite H1AH treatment, and there have historically been limited alternatives available for this population.

“[Dupilumab] 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,” George D. Yancopoulos, MD, PhD, board co-chair, president, and chief scientific officer at Regeneron, said in a statement.¹ “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, [dupilumab], has the potential to change yet another treatment paradigm.”

The pediatric approval is supported primarily by data from the LIBERTY-CUPID phase 3 program. Key evidence was drawn from 2 replicate, randomized, double-blind, placebo-controlled trials—Study A and Study C (NCT04180488)—conducted in patients 6 years and older with CSU who were antihistamine-refractory and anti-immunoglobulin E (IgE)–naive. In both trials, dupilumab significantly reduced itch severity and urticaria activity (as measured by the weekly Urticaria Activity Score [UAS7], a composite of itch and hives) compared with placebo at week 24.¹ Dupilumab also improved rates of well-controlled disease (UAS7 ≤ 6) and complete response (UAS7 = 0) vs placebo.

Pharmacokinetic data from the phase 3, single-arm CUPIDKids study (NCT05526521) in children aged 2 to 11 years with CSU supported the extrapolation of efficacy and safety to the younger age group. Safety data from Study B (NCT04180488), which enrolled patients 12 years and older who had not responded to or were intolerant to anti-IgE therapy, provided additional safety evidence, and findings from pediatric patients in other dupilumab indications further informed the safety profile in this age group.

Across all 4 LIBERTY-CUPID studies, the safety profile of dupilumab was generally consistent with its profile in other approved dermatological indications. Injection site reactions were the most common adverse reaction (≥2%) observed more frequently with dupilumab than placebo. No new safety signals were identified in children aged 2 to 11 years with CSU.

In children aged 2 to 5 years, dupilumab is administered at 200 mg subcutaneously every 4 weeks for those weighing at least 5 kg to less than 15 kg and 300 mg every 4 weeks for those weighing at least 15 kg to less than 30 kg, without an initial loading dose. For children aged 6 to 11 years, weight-based dosing follows the same regimens as for older pediatric patients, with an initial loading dose.

In addition to this US approval, dupilumab was approved in the European Union for CSU in children aged 2 to 11 years in April 2026, as well as other other countries.

“Children with uncontrolled chronic spontaneous urticaria continue to experience the unpredictable appearance of debilitating itch and hives,” Alyssa Johnsen, MD, PhD, global therapeutic area head of immunology development at Sanofi, said in a statement.¹ “Until now, these patients had to rely on limited treatment options that didn’t address potential critical mediators of chronic spontaneous urticaria. Dupixent is the first biologic approved for patients as young as 2 years of age, offering a targeted approach that inhibits IL [interleukin]-4 and IL-13 signaling, 2 key and central drivers of the type 2 inflammation that contributes to this disease. Today’s approval underscores our ongoing commitment to advancing therapies for young patients with significant unmet needs.”

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. April 22, 2026. Accessed April 23, 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. Santoro A. FDA greenlights dupilumab for chronic spontaneous urticaria, marking first approval in a decade. AJMC^®. April 18, 2025. Accessed April 23, 2026. https://www.ajmc.com/view/fda-greenlights-dupilumab-for-chronic-spontaneous-urticaria-marking-first-approval-in-a-decade