FDA approves dupilumab for chronic spontaneous urticaria in ages 12 and up
The sBLA for dupilumab (Dupixent) was supported by data from the phase 3 LIBERTY-CUPID clinical program in patients with uncontrolled CSU.
FDA approves dupilumab for chronic spontaneous urticaria in ages 12 and up | Image Credit: © Calin - © Calin - stock.adobe.com.
On April 18, 2025, the FDA approved a resubmitted supplemental biologics license application (sBLA) for dupilumab (Dupixent; Regeneron and Sanofi) to treat chronic spontaneous urticaria (CSU) in adults and pediatric patients aged 12 years and older, according to a press release from Regeneron.1
The interleukin (IL)-4 and IL-13 inhibitor for this CSU indication is designed to treat those whose disease is not adequately controlled with H1 antihistamine treatment, and is the first targeted therapy for CSU in over a decade.1,2
With the federal agency's decision, CSU is now the 7th disease with underlying type 2 inflammation in which dupilumab is approved.
"[Dupilumab] is the first new targeted treatment for CSU, in over ten years, with pivotal trials demonstrating its ability to help patients significantly reduce the hallmark symptoms of intense itch and unpredictable hives associated with this disease,” said George D. Yancopoulos, MD, PhD, in a statement. Yancopoulos is the board co-chair, president, and chief scientific officer at Regeneron. "With this FDA decision, Dupixent is now approved for seven chronic, debilitating atopic conditions driven in part by underlying type 2 inflammation, several of which have been shown to co-morbidly occur with CSU, such as atopic dermatitis and asthma – providing patients with one treatment that might help multiple atopy conditions. We look forward to bringing Dupixent to the more than 300,000 CSU patients in the U.S. with inadequately controlled disease on standard-of-care treatment who, until now, had limited treatment options," added Yancopoulos.1
Data that led to dupilumab approval for CSU
Acceptance of the sBLA was based on data from the LIBERTY-CUPID phase 3 clinical program that featured Study A, Study B, and Study C. This sBLA added results from Study C, which was carried out in patients with uncontrolled CSU who were on standard-of-care antihistamines. This study met primary and key secondary endpoints that confirmed results in Study A.2
Study A and Study B were phase 3, randomized, placebo-controlled, double-blind trials, that compared dupilumab with placebo in symptomatic CSU patients despite H1-AH—the treatment met the primary and all key secondary outcomes. According to data previously reported by Contemporary Pediatrics, Study A was made up of omalizumab-naive patients aged 6 years and up (n = 138) while Study B featured omalizumab-intolerant/incomplete responders aged 12 years and older (n = 138).
Both Weekly Urticaria Activity Score (UAS7) and Weekly Itch Severity Score (ISS7) improved with dupilumab compared to treatment in Study A, according to data published in The Journal of Allergy and Clinical Immunology ((difference -8.5 [95% CI, -13.2 to -3.9; (difference -8.5 [95% CI, -13.2 to -3.9; P = .0003] and -4.2 [95% CI, -6.6 to -1.8; P = .0005]).3
Trial investigators noted that in Study B, UAS7 improved (difference -5.8 [95% CI, -11.4 to -0.3; P = .0390]), with a numerical trend in ISS7 (difference -2.9 [95% CI, -5.7 to -0.07; nominal P = .0449, not significant]). Pooled safety data across each study revealed consistency between dupilumab and placebo, which aligned with the known safety profile of dupilumab.
Dupilumab improves itch and hives in CSU patients
The chronic inflammatory skin disease is characterized by the sudden onset of hives on the skin or swelling under the skin. Study C enrolled 151 children and adults to receive either dupilumab (n = 74) or placebo (n = 77), added to standard-of-care histamine-1 (H1) antihistamines, Sanofi previously announced. At 24 weeks, efficacy for patients receiving dupilumab, compared to placebo, was:4
- 8.64-point reduction in itch severity from baseline with dupilumab versus a 6.10-point reduction with placebo (P = 0.02)
- 15.86-point reduction in urticaria activity (itch and hive) severity from baseline with dupilumab vs an 11.21-point reduction with placebo (P = 0.02)
- 30% of dupilumab-treated patients reported no urticaria, or complete response, compared to individuals on placebo (P = .02).
References:
1. Dupixent (dupilumab) Approved in the US as the First New Targeted Therapy in Over a Decade for Chronic Spontaneous Urticaria (CSU). Regeneron. Press release. April 18, 2025. Accessed April 18, 2025. https://newsroom.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-approved-us-first-new-targeted-therapy-over
2. Fitch, J. FDA accepts application for dupilumab to treat chronic spontaneous urticaria. Contemporary Pediatrics. November 15, 2024. Accessed April 17, 2025. https://www.contemporarypediatrics.com/view/fda-accept-application-for-dupilumab-to-treat-chronic-spontaneous-urticariaContemporary Pediatrics. November 15, 2024. Accessed April 17, 2025. https://www.contemporarypediatrics.com/view/fda-accept-application-for-dupilumab-to-treat-chronic-spontaneous-urticaria
3. Maurer M, Casale TB, Saini SS, et al. Dupilumab in patients with chronic spontaneous urticaria (LIBERTY-CSU CUPID): Two randomized, double-blind, placebo-controlled, phase 3 trials. J Allergy Clin Immunol. 2024 Jul;154(1):184-194. doi:10.1016/j.jaci.2024.01.028. Epub 2024 Feb 29. PMID: 38431226
4. Press Release: Dupixent phase 3 study confirms significant improvements in itch and hives for patients with CSU. Sanofi. Press release. September 11, 2024. Accessed April 17, 2025. https://www.sanofi.com/en/media-room/press-releases/2024/2024-09-11-05-05-00-2944239
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