FDA Approves Dupilumab for Treatment of Bullous Pemphigoid in Adults

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The US FDA approved dupilumab (Dupixent®; Sanofi, Regeneron) as the first targeted treatment for adults with bullous pemphigoid (BP). The approval is based on positive clinical trial findings from the ADEPT phase 2/3 clinical trial and marks the 8th indication for dupilumab in a disease with underlying type 2 inflammation.

“People affected by bullous pemphigoid endure unrelenting itch and painful blisters that can damage the skin. Until now, these primarily elderly patients have had limited therapeutic options available, with potential side effects that have often added to their burden,” Patrick Dunn, executive director, International Pemphigus and Pemphigoid Foundation, said in a June 20, 2025, press release. “The approval of Dupixent for bullous pemphigoid brings a novel treatment approach to patients and their caregivers.”

The FDA granted the approval following a priority review and had previously designated dupilumab an orphan drug for BP, a rare condition affecting fewer than 200 000 Americans. Additional regulatory applications for the indication are under review in the European Union, Japan, and China.

“By addressing two central drivers of the underlying type 2 inflammation that contributes to bullous pemphigoid, Dupixent is the first targeted medicine to allow patients the potential to achieve sustained remission and reduce itch,” Alyssa Johnsen, MD, PhD, global therapeutic area head, immunology and oncology development at Sanofi, said in the press announcement.

The randomized, double-blind, placebo-controlled ADEPT trial enrolled 106 adults with moderate-to-severe BP. Participants were randomized to receive either dupilumab 300 mg every 2 weeks (n = 53) or placebo (n = 53), both in combination with standard-of-care oral corticosteroids. Corticosteroids were tapered beginning between weeks 4 and 6 in patients achieving disease control, with the goal of complete discontinuation by week 16.

At week 36, 18.3% of dupilumab-treated patients achieved sustained disease remission compared with 6.1% in the placebo group—a 12.2% absolute difference (95% CI, –0.8 to 26.1), the trial’s primary endpoint. Additionally, 38.3% of patients in the dupilumab group experienced a clinically meaningful reduction in itch, defined as a ≥4-point improvement in Peak Pruritus Numerical Rating Scale score, compared with 10.5% in the placebo group. The cumulative median dose of oral corticosteroids was also lower in the dupilumab group (2.8 g) than in the placebo group (4.1 g) by week 36.

Adverse events reported more frequently in the dupilumab group (≥2%) included arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis. One case of acute generalized exanthematous pustulosis occurred in the treatment group. Dupilumab is administered as a subcutaneous injection and, for BP, is dosed at 300 mg every two weeks following a loading dose.

“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,” George D. Yancopoulos, MD, PhD, board cochair, president, and chief scientific officer at Regeneron, and a principal inventor of dupilumab, said in the press release. “Additionally, this approval further reinforces the demonstrated safety profile of Dupixent in a broad age range of patients, from infants to elderly people, and across dermatological, respiratory, and gastrointestinal diseases.”

Source: Press Release: Dupixent approved in the US as the only targeted medicine to treat patients with bullous pemphigoid. News release. Sanofi. June 20, 2025. Accessed June 20, 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-06-20-05-00-00-3102518