The FDA has approved Regeneron’s and Sanofi’s Dupixent (dupilumab) as the first and only targeted therapy for adult patients with bullous pemphigoid (BP), a rare and chronic autoimmune blistering skin disease that primarily affects the elderly. The approval was based on results from the Phase II/III ADEPT trial, in which Dupixent demonstrated clinically meaningful benefits, including a higher rate of sustained disease remission, greater itch reduction compared to placebo, and a lower median cumulative oral corticosteroid (OCS) dose.¹

How Did Dupixent Perform in the ADEPT Trial to Secure FDA Approval for Bullous Pemphigoid?

“People affected by bullous pemphigoid endure unrelenting itch and painful blisters that can damage the skin,” said Patrick Dunn, executive director, International Pemphigus and Pemphigoid Foundation, in a press release. “Until now, these primarily elderly patients have had limited therapeutic options available, with potential side effects that have often added to their burden. The approval of Dupixent for bullous pemphigoid brings a novel treatment approach to patients and their caregivers, and we are grateful for the tireless efforts of the scientific community who helped us reach this critical milestone.”

Trial Design and Methodology

• The double-blind, placebo-controlled ADEPT trial evaluated the efficacy and safety of Dupixent in 106 adults with moderate-to-severe BP for 52 weeks.
• Patients were randomly assigned to receive either Dupixent or placebo every two weeks following an initial loading dose, in combination with standard-of-care OCS.
• After two weeks of sustained disease control, patients began tapering OCS with the goal of completing the taper by week 16; they then continued on Dupixent or placebo alone for at least 20 weeks unless rescue treatment was required.
• The primary endpoint of the trial was the proportion of patients achieving sustained disease remission at 36 weeks.
• Secondary endpoints included the proportion of patients with ≥4-point reduction in Peak Pruritus Numerical Rating Scale and total cumulative OCS dose.

Efficacy Results

• Dupixent achieved an 18.3% sustained disease remission rate versus 6.1% in the placebo group.
• A clinically meaningful itch reduction was observed in 38.3% of the Dupixent cohort compared to 10.5% in the placebo group.

The median cumulative OCS dose was 2.8 grams in the Dupixent group versus 4.1 grams in the placebo group.

Safety Profile

• Common adverse events associated with Dupixent included arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis.
• One case of acute generalized exanthematous pustulosis was reported in the Dupixent group.
• The safety profile of Dupixent was reported to be consistent with prior indications of the treatment.¹

“Today’s approval extends the remarkable ability of Dupixent to transform treatment paradigms for people living with a variety of diseases with underlying type 2 inflammation, from common conditions like asthma and atopic dermatitis, to rarer ones such as eosinophilic esophagitis and prurigo nodularis, and now including bullous pemphigoid,” said George D. Yancopoulos, MD. PhD, board co-chair, president, chief scientific officer, Regeneron, and a principal inventor of Dupixent, in the press release. “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. 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.”

Epidemiology and Outlook

According to Cleveland Clinic, the global prevalence of BP is between 2.4 and 23 cases per one million people in the general population. In people aged 80 years and older, the prevalence is between 190 and 312 cases per one million people.²

“Until now, treating bullous pemphigoid was very challenging for elderly patients struggling with the debilitating impact of blisters and lesions, and potentially co-morbid conditions,” said Alyssa Johnsen, MD, PhD, global therapeutic area head, immunology and oncology development, Sanofi, in the press release. “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. This approval in the U.S. is important for the thousands of patients living with bullous pemphigoid, and we look forward to working with regulators around the world to bring this innovative medicine to more patients in need.”

References

Dupixent® (dupilumab) Approved in the U.S. as the Only Targeted Medicine to Treat Patients with Bullous Pemphigoid (BP). Regeneron. June 20, 2025. Accessed June 20, 2025. https://investor.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-approved-us-only-targeted-medicine-treat

Bullous Pemphigoid. Cleveland Clinic. June 20, 2025. Accessed June 20, 2025. https://my.clevelandclinic.org/health/diseases/15855-bullous-pemphigoid