Dupilumab Proves Benefit in Bullous Pemphigoid Ahead of June 2025 FDA Decision

Data from the 2025 American Academy of Dermatology (AAD) Annual Meeting suggest use of dupilumab (Dupixent) was associated with significant improvements in disease activity and burden among patients with bullous pemphigoid.

Coming just weeks after Regeneron and Sanofi announced the US Food and Drug Administration's acceptance of their supplemental Biologics License Application for dupilumab in bullous pemphigoid, the full results of LIBERTY-BP ADEPT provide key insights into the potential of dupilumab among this patient population.1,2

“People with bullous pemphigoid live with unrelenting itch, blisters, and painful lesions that can be debilitating and make it difficult to function daily. Moreover, current treatment options can be challenging for this primarily elderly patient population because they work by suppressing their immune system,” said principal investigator Victoria Werth, MD, chief of the Division of Dermatology at the Philadelphia Veterans Administration Hospital and professor of Dermatology and Medicine at the Hospital of the University of Pennsylvania and the Veteran's Administration Medical Center, in a statement.2

Few agents have carved a role for itself in as many disease states as dupilumab. Boasting approvals for more than half a dozen indications since its initial approval for atopic dermatitis in 2017, the agent now seems poised to add another indication for bullous pemphigoid to its ongoing list. In September 2024, Regeneron and Sanofi announced positive topline data from the trial, which suggested the trial met the primary and all key secondary endpoints in adults with moderate-to-severe disease, with approximately 5 times more patients achieved sustained disease remission with dupilumab than placebo.3,4

A phase 2/3 trial, LIBERTY-BP ADEPT was designed as a double-blind, placebo-controlled trial to examine the efficacy and safety of dupilumab among 106 adults with moderate-to-severe bullous pemphigoid for a 52-week treatment period. These patients were randomized in a 1:1 ratio to either dupilumab 300 mg every two weeks following an initial loading dose or placebo therapy added to standard of care oral corticosteroids.1,2

The primary outcome of interest for the trial was the proportion of patients achieving sustained disease remission at 36 weeks. Per trial protocol, patients underwent an oral corticosteroid tapering regimen if control of disease activity was maintained.1,2

Of note, investigators defined sustained remission as complete clinical remission with completion of OCS taper by 16 weeks without relapse and no rescue therapy use during the 36-week treatment period while relapse was defined as appearance of 3 or more new lesions a month or 1 or more large lesion or urticarial plaque that did not heal within a week.1,2

Upon analysis, 36-week data from the trial indicated 20% of patients receiving dupilumab experienced sustained disease remission relative to 4% (P = .0114) among the placebo group. Additionally, at week 36, 40% achieved a 90% or greater reduction in disease severity compared to 10% with placebo (P = .0003) and 40% achieved clinically meaningful itch reduction compared to 11% with placebo (P = .0006). Further analysis suggested use of dupilumab was associated with a 1678 mg mean reduction in cumulative oral corticosteroids exposure (P = .022) and a 54% reduction in risk of rescue medication use (P = .0016).1,2

Safety analysis of the trial revealed overall rates of adverse events of 96% for both dupilumab and placebo. Investigators pointed out adverse events observed more commonly with dupilumab relative to placebo in at least 3 patients included peripheral edema (n=8 vs n=5), arthralgia (n=5 vs n=3), back pain (n=4 vs n=2), blurred vision (n=4 vs n=0), hypertension (n=4 vs n=3), asthma (n=4 vs n=1), conjunctivitis (n=4 vs n=0), constipation (n=4 vs n=1), upper respiratory tract infection (n=3 vs n=1), limb injury (n=3 vs. n=2), and insomnia (n=3 vs n=2). However, investigators underlined there were no events leading to death in the dupilumab group whereas 2 adverse events leading to death occurred in the placebo group.1,2

“By targeting the underlying type 2 inflammation, which is a key driver for bullous pemphigoid, [dupilumab] is the first investigational biologic to show sustained disease remission and reduce disease severity and itch compared to placebo in a clinical study,” Werth added.2

References:

1. Werth V. Efficacy and Safety of Dupilumab in Patients With Bullous Pemphigoid: Results From LIBERTY-BP ADEPT Phase 2/3 Study. Presented at the 2025 American Academy of Dermatology (ADA) Annual Meeting. Orlando, FL. March 07-11, 2025.

2. Sanofi. Press release: Dupixent is the first and only biologic to achieve significant improvements in disease remission and symptoms in bullous pemphigoid positive pivotal study. Sanofi. September 11, 2025. Accessed March 8, 2025. https://www.sanofi.com/en/media-room/press-releases/2024/2024-09-11-05-00-00-2944237.

3. Stewart J. Dupixent (dupilumab) FDA approval history. Drugs.com. October 1, 2024. Accessed March 8, 2025. https://www.drugs.com/history/dupixent.html.

4. Sanofi. Press release: Dupixent is the first and only biologic to achieve significant improvements in disease remission and symptoms in bullous pemphigoid positive pivotal study. Sanofi. September 11, 2025. Accessed March 8, 2025. https://www.sanofi.com/en/media-room/press-releases/2024/2024-09-11-05-00-00-2944237.