Dupixent Makes History with FDA Approval as First New CSU Treatment in Over a Decade

The U.S. Food and Drug Administration (FDA) approved Sanofi’s dupilumab (Dupixent) for the treatment of chronic spontaneous urticaria (CSU) in patients aged 12 and older whose condition remains uncontrolled with H1-antihistamines. This approval represents the first new targeted therapy for CSU in over ten years. Regulatory authorities in Japan, the United Arab Emirates, and Brazil have approved Dupixent for chronic spontaneous urticaria (CSU). Regulatory submissions are under review in additional regions, including the European Union.

Approval of Dupixent Backed by Positive Phase 3 Trial Results Showing Significant Symptom Relief

The FDA’s approval of Dupixent for CSU is supported by findings from two phase 3 trials, Study A and Study C, which enrolled biologic-naïve patients aged 12 and older whose symptoms persisted despite antihistamine use. These studies evaluated Dupixent as an add-on to standard antihistamine therapy compared to antihistamines alone. Both trials achieved their primary and key secondary endpoints, with Dupixent showing greater reductions in itch severity and overall urticaria activity, including both itch and hives, at 24 weeks. 

Additionally, patients treated with Dupixent were more likely to achieve well-controlled disease or a complete response compared to those receiving placebo. Study B, which included patients who had an inadequate response to or could not tolerate anti-IgE therapy, contributed further safety data. Across Studies A, B, and C, the safety profile of Dupixent remained consistent with that observed in its previously approved indications.

In the pooled safety analysis, the most common adverse event (occurring in 2% or more of patients) seen more frequently in the Dupixent group than in the placebo group was injection site reactions. Dupilumab works by inhibiting the signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two key pathways involved in inflammation, thereby helping to alleviate symptoms of CSU and reduce severe itching. 

Sanofi has already approved the medication for several other inflammatory and allergic conditions, including atopic dermatitis, asthma, chronic obstructive pulmonary disease (COPD), and more. George D. Yancopoulus, M.D., Ph.D, board co-Chair, President and Chief Scientific Officer at Regeneron, stated, “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.”

CSU Affects 10-31% with Mental Health Issues and 20% with Autoimmune Thyroiditis

CSU can affect individuals of any age but occurs most frequently in women between 30 and 50 years old. Diagnosis is primarily clinical, based on the presence of spontaneously recurring wheals, angioedema, or both. In most cases, CSU persists for over a year, either as a single episode or through repeated occurrences, and is often associated with comorbidities such as chronic inducible urticaria (over 10%), autoimmune thyroiditis (around 20%), metabolic syndrome (6%–20%), and mental health conditions like anxiety (10%–31%) and depression (7%–29%). More than half of CSU cases involve autoimmune endotypes mediated by mast cell–activating IgE and/or IgG autoantibodies.

CSU has a significant impact on quality of life, with approximately 40% of patients reporting a Dermatology Life Quality Index (DLQI) score above 10, indicating a very large to extremely large negative effect. Current CSU treatments beyond H1-antihistamines and omalizumab, such as cyclosporine, corticosteroids, and leukotriene receptor antagonists, often show limited effectiveness and come with safety concerns. 

Many patients continue to suffer from uncontrolled symptoms due to poor response or intolerable side effects. The FDA’s approval of Dupixent for represents a critical advancement. By targeting IL-4 and IL-13 signaling, dupilumab offers a new, targeted treatment option with a well-established safety profile, providing much-needed relief for patients who have exhausted standard therapies.

Alyssa Johnsen, M.D., Ph.D., Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi, stated, “CSU patients with uncontrolled disease experience highly burdensome itch and hives that can significantly disrupt daily living. This FDA approval provides a new treatment option to help address the underlying drivers of these severe and recurring signs and symptoms. Dupixent has the potential to improve outcomes for CSU patients who previously had limited treatment options.”

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Author

Denisse Sandoval