The U.S. Food and Drug Administration (FDA) has approved dupilumab (Dupixent) for the treatment of adult and adolescent patients with chronic spontaneous urticaria (CSU) who remain symptomatic despite H1 antihistamine treatment. This approval marks a significant advancement for patients with this debilitating skin condition and represents the sixth FDA-approved indication for dupilumab, jointly developed by Sanofi and Regeneron Pharmaceuticals.
Chronic spontaneous urticaria affects approximately 300,000 people in the United States alone, characterized by the sudden appearance of itchy hives, angioedema (swelling), or both, lasting for more than six weeks with no identifiable external trigger. The condition can significantly impact quality of life, with many patients experiencing symptoms for years.
Breakthrough in Urticaria Treatment
The approval is based on results from the Phase 3 LIBERTY-CUPID clinical trial program, which demonstrated that dupilumab significantly reduced itch and hives in patients with CSU compared to placebo. The trial included patients who remained symptomatic despite standard-of-care treatment with H1 antihistamines.
"This approval represents an important milestone for patients with chronic spontaneous urticaria who have had limited treatment options," said Dr. Marcus Maurer, Professor of Dermatology and Allergy at Charité – Universitätsmedizin Berlin and principal investigator of the clinical trial. "Many patients continue to experience debilitating symptoms despite current therapies. Dupilumab offers a new approach by targeting key inflammatory pathways involved in this condition."
Novel Mechanism of Action
Dupilumab is a fully human monoclonal antibody that inhibits the signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two key cytokines involved in type 2 inflammation. This mechanism differs from existing biological treatments for CSU, which primarily target IgE or its receptor.
"The approval of dupilumab for CSU provides physicians with a new treatment option that addresses the underlying type 2 inflammation driving symptoms in many patients," said Dr. George D. Yancopoulos, President and Chief Scientific Officer at Regeneron. "This represents the sixth indication for dupilumab, further validating the importance of IL-4 and IL-13 as drivers of multiple allergic and inflammatory conditions."
Clinical Trial Results
In the pivotal Phase 3 study, patients receiving dupilumab 300 mg every two weeks experienced:
- 63% reduction in itch severity at 24 weeks (primary endpoint) compared to 35% with placebo
- 65% reduction in urticaria activity score (UAS7) at 24 weeks compared to 37% with placebo
- Significant improvements in quality of life measures
- Rapid onset of action, with improvements observed as early as week 1
The safety profile was consistent with the known safety profile of dupilumab in its approved indications, with the most common side effects including injection site reactions, conjunctivitis, and upper respiratory tract infections.
Expanding Treatment Landscape
Prior to this approval, treatment options for CSU were limited. First-line therapy typically consists of H1 antihistamines, often at higher-than-standard doses. For patients who don't respond adequately, omalizumab (an anti-IgE monoclonal antibody) has been the only approved biological therapy.
"Despite available treatments, many patients with chronic spontaneous urticaria continue to struggle with persistent symptoms that significantly impact their daily lives," said Dr. Sandra Waserman, Professor of Medicine at McMaster University. "Having a new biological option with a different mechanism of action is a welcome addition to our therapeutic arsenal."
Commercial Implications
For Sanofi and Regeneron, this approval further strengthens dupilumab's position as a versatile immunomodulatory therapy. The drug has previously received FDA approval for atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, and prurigo nodularis.
"The approval of Dupixent for CSU represents another important achievement in our mission to address the needs of patients with type 2 inflammatory diseases," said Bill Sibold, Executive Vice President of Specialty Care at Sanofi. "We are committed to ensuring that eligible patients can access this important new treatment option."
Patient Access and Support
Sanofi and Regeneron have announced that they will work with healthcare providers, payers, and patient advocacy groups to help ensure appropriate patients can access dupilumab. The companies' patient support program, DUPIXENT MyWay, will provide resources to patients, including insurance benefit verification, prior authorization support, and financial assistance for eligible patients.
The companies expect dupilumab to be available for CSU patients in the coming weeks, with a recommended dose of 300 mg administered subcutaneously every two weeks. The therapy can be self-administered after proper training by a healthcare professional.
This approval provides a valuable new option for dermatologists, allergists, and immunologists treating patients with this challenging condition, particularly for those who have not responded adequately to existing therapies.
