FDA Accepts sBLA for Dupilumab as Potential BP Treatment

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Regeneron Pharmaceuticals, Inc. and Sanofi announced today that the US Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for dupilumab (Dupixent) for priority review as a potential treatment for adults with bullous pemphigoid (BP). If approved, dupilumab would become the first targeted therapy available for BP in the United States. According to the companies, the FDA's decision is expected by June 20, 2025.¹

Clinical Trial and Efficacy Data

The sBLA for dupilumab is supported by data from a pivotal phase 2/3 clinical study involving 106 adults with moderate to severe BP. The primary endpoint was sustained disease remission, defined as complete clinical remission following the tapering and discontinuation of oral corticosteroids (OCS) by week 16, and without relapse and no rescue therapy use during the 36-week treatment period. Researchers stated patients treated with dupilumab demonstrated a 5-fold improvement in achieving sustained disease remission compared to those receiving placebo. Furthermore, they found dupilumab significantly reduced disease severity, itch, and overall corticosteroid use during the study period.²

Safety and Adverse Events

The clinical trial data also provided insight into the safety profile of dupilumab. Adverse events more frequently observed in patients receiving dupilumab (compared to placebo) included peripheral edema, arthralgia, back pain, blurred vision, hypertension, asthma, conjunctivitis, constipation, upper respiratory tract infection, limb injury, and insomnia. These findings underscore the need for continued monitoring of the drug’s safety in this patient population.

Regulatory Status and Priority Review

The FDA grants priority review status to regulatory applications that present therapies with the potential to significantly improve treatment outcomes for serious or life-threatening conditions. Dupilumab had previously received orphan drug designation for BP, signifying its intended use in treating a rare disease affecting fewer than 200,000 individuals in the US. The safety and efficacy of dupilumab for BP remain under clinical evaluation and have not yet been reviewed by any regulatory authority.

Clinical Significance

BP primarily affects elderly individuals and presents with intense pruritus, widespread blisters, skin reddening, and painful lesions. In severe cases, the condition leads to significant morbidity, impacting daily activities and increasing susceptibility to infections. Current treatment options rely heavily on systemic corticosteroids and immunosuppressive therapies, which carry risks of adverse effects, particularly in older patients. It is estimated that approximately 27,000 adults in the US live with BP that is inadequately controlled by corticosteroids.

Mechanism of Action of Dupilumab

Dupilumab is a fully human monoclonal antibody that targets and inhibits interleukin-4 and interleukin-13 signaling pathways. These pathways are critical mediators of type 2 inflammation, which plays a central role in BP and other inflammatory conditions. Notably, Dupixent is not classified as an immunosuppressant, distinguishing it from traditional steroid-based treatments.

Broader Clinical Development of Dupilumab

Dupilumab has been evaluated in over 60 clinical trials involving more than 10,000 patients with type 2 inflammatory diseases. It has received regulatory approval in over 60 countries for multiple indications, including atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous urticaria, and chronic obstructive pulmonary disease.

In addition to BP, ongoing phase 3 studies are investigating dupilumab in other dermatologic and inflammatory conditions such as chronic pruritus of unknown origin and lichen simplex chronicus. These indications remain under clinical evaluation, with safety and efficacy data pending regulatory review.

Conclusion

The FDA’s acceptance of the sBLA for dupilumab represents a potential milestone in the treatment of BP. If approved, it would provide the first targeted therapy for BP, offering an alternative to traditional systemic corticosteroids. The decision, expected by mid-2025, will determine whether the drug can be a new standard of care for BP patients struggling with current treatment limitations. Continued research and clinical assessments will be crucial in establishing the long-term benefits and safety profile of dupilumab in this indication.

References

1. Dupixent sBLA accepted for FDA priority review for the targeted treatment of bullous pemphigoid. News Release. Sanofi. Published February 18, 2025. Accessed February 18, 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-02-18-06-00-00-3027482
2. Murrell DF, Joly P, Werth VP, et al. Study design of a phase 2/3 randomized controlled trial of dupilumab in adults with bullous pemphigoid: LIBERTY-BP ADEPT. Adv Ther. 2024 Jul;41(7):2991-3002. doi: 10.1007/s12325-024-02810-3.