FDA Approves Dupilumab as First Targeted Treatment for Bullous Pemphigoid in Adults

On June 20, 2025, the US Food and Drug Administration (FDA) approved dupilumab (Dupixent) for the treatment of adult patients with bullous pemphigoid (BP), making it the first and only targeted therapy approved for this rare, chronic autoimmune blistering skin disease.

“Until now, these primarily elderly patients [with BP] have had limited therapeutic options available, with potential side effects that have often added to their burden,” said Patrick Dunn, executive director of the International Pemphigus and Pemphigoid Foundation. “The approval of Dupixent for BP brings a novel treatment approach to patients and their caregivers.”

The FDA approval is based on data from the pivotal phase 2/3 ADEPT study, which evaluated dupilumab in 106 adults with moderate-to-severe BP. Patients were randomized to receive dupilumab 300 mg (n=53) or placebo (n=53), in addition to standard-of-care oral corticosteroids. All patients followed a standardized corticosteroid tapering protocol as disease control allowed.

At week 36, the study demonstrated:

• 18.3% of patients receiving dupilumab achieved sustained disease remission vs 6.1% on placebo
• 38.3% achieved clinically meaningful itch reduction vs 10.5% on placebo
• Median cumulative corticosteroid dose was reduced to 2.8 g vs 4.1 g with placebo

Sustained disease remission was defined as complete clinical remission with successful corticosteroid taper by week 16 and no relapse or rescue therapy through week 36.

The most common adverse events (≥2%) reported more frequently with dupilumab included arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis. Also, 1 patient treated with dupilumab experienced acute generalized exanthematous pustulosis. Overall, the safety profile of dupilumab was consistent with that seen in other approved indications.

“Dupixent has shown the potential to improve the most challenging effects of bullous pemphigoid, while helping some patients achieve sustained disease remission and decreased oral corticosteroid use,” said George D. Yancopoulos, MD, PhD.

With this latest indication, dupilumab is now approved in the United States to treat 8 distinct diseases driven by type 2 inflammation, including conditions of the skin, lungs, and gastrointestinal tract. The approval was granted under priority review and follows the FDA’s earlier Orphan Drug Designation for dupilumab in BP.

Dupixent is administered as a 300 mg subcutaneous injection every 2 weeks following an initial loading dose and may be given in a clinical setting or at home after appropriate training by a health care professional.

Reference

Dupixent approved in the US as the only targeted medicine to treat patients with bullous pemphigoid. Press release. Sanofi. June 20, 2025. Accessed June 20, 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-06-20-05-00-00-310251