FDA Prioritizes Review of Dupixent® sBLA for Bullous Pemphigoid Treatment

Regeneron Pharmaceuticals, Inc. and Sanofi have announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) for the treatment of adults with bullous pemphigoid (BP). A decision from the FDA is anticipated by June 20, 2025. If approved, Dupixent would become the first and only targeted therapy for BP in the United States.

Bullous pemphigoid is a chronic and distressing skin condition driven by type 2 inflammation, primarily affecting the elderly. It is characterized by severe itching, blisters, skin reddening, and painful lesions. These symptoms can cover large areas of the body, leading to bleeding, crusting, and increased susceptibility to infections, significantly impacting patients' daily lives. In the United States, approximately 27,000 adults suffer from BP that cannot be controlled by systemic corticosteroids.

The Priority Review status was granted following positive results from a pivotal clinical trial evaluating Dupixent's efficacy and safety in 106 adults with moderate-to-severe BP. The study's primary endpoint was achieved with patients treated with Dupixent exhibiting a fivefold increase in sustained disease remission compared to those on placebo. Sustained disease remission was defined as complete clinical remission and successful tapering off oral corticosteroids by week 16, with ongoing treatment using only Dupixent for a minimum of 20 weeks, without any relapse or the need for rescue therapy during the 36-week treatment period. Additionally, the trial demonstrated that Dupixent significantly reduced disease severity, itching, and corticosteroid use compared to placebo.

The most common adverse events observed in patients treated with Dupixent, compared to placebo, included peripheral edema, arthralgia, back pain, blurred vision, hypertension, asthma, conjunctivitis, constipation, upper respiratory tract infections, limb injury, and insomnia.

Dupixent works by inhibiting the signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two key drivers of type 2 inflammation, without acting as an immunosuppressant. This monoclonal antibody was developed using Regeneron's proprietary VelocImmune® technology. The Dupixent development program has demonstrated significant clinical benefits across multiple conditions associated with type 2 inflammation, such as atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, and more.

Regeneron and Sanofi are also exploring the potential of Dupixent in treating other diseases driven by type 2 inflammation, such as chronic pruritus of unknown origin, lichen simplex chronicus, and additional potential indications. These uses are currently under clinical investigation, and their safety and efficacy have yet to be fully evaluated by regulatory authorities.

In the United States, Dupixent is approved for the treatment of several conditions, including moderate-to-severe atopic dermatitis in adults and children aged six months and older, eosinophilic or steroid-dependent asthma in individuals aged six years and older, and chronic rhinosinusitis with nasal polyps in those aged 12 years and older. It is also approved for treating eosinophilic esophagitis in patients aged one year and older and prurigo nodularis in adults.

The regulatory journey of Dupixent highlights the ongoing commitment of Regeneron and Sanofi to advance therapeutic options for patients with serious diseases, providing hope and potential relief for those affected by chronic conditions such as bullous pemphigoid.