FDA Grants Priority Review for Dupilumab in Adults with Bullous Pemphigoid

The Sanofi - Aventis Groupe has announced that the US Food and Drug Administration (FDA) granted priority review status to the supplemental biologics license application (sBLA) for dupilumab (Dupixent) therapy among adult patients with bullous pemphigoid.¹

The February 18 announcement highlights that dupilumab will receive an expedited review process, noting that the FDA had previously given it the orphan drug designation for bullous pemphigoid. The condition itself is a chronic autoimmune skin condition that is known to mostly impact older patients and is driven by type-2 inflammation.

The skin disease is estimated to impact around 27,000 adults for whom systemic corticosteroids do not lead to an adequate treatment response. Bullous pemphigoid is characterized by severe widespread redness, itching, and blisters, potentially resulting in effects such as crusting and skin damage that can affect the quality of life of those with the disease.

Dupilumab is a fully human monoclonal antibody designed to inhibit the interleukin-4 (IL-4) and interleukin-13 (IL-13) signaling pathways, as these 2 are known to be key drivers of type-2 inflammation. Dupilumab is slightly different from traditional immunosuppressants, as it reduces inflammation without broadly suppressing patients’ immune systems.

The drug’s mechanism of action has, in recent 3 clinical research, mitigated the type-2 inflammatory responses in many patients. The recent sBLA submission to the FDA was based on recent results from a pivotal clinical study assessing dupilumab’s efficacy and safety among 106 adults with moderate-to-severe bullous pemphigoid.²

Overall, this clinical trial was shown by the team to have achieved its main endpoint, with the results indicating that individuals treated with dupilumab were 5 times more likely to experience sustained disease remission as opposed to trial participants in the placebo arm.^1,2 The investigative team had defined sustained remission as the attainment among patients of complete clinical remission and the successful tapering off of oral corticosteroids by the 16-week mark.

The latter endpoint would mean that participants would remain on dupilumab monotherapy for at least 20 weeks without relapsing or the need for rescue therapy throughout the 36-week study period. Substantial reductions in patients’ itch, disease severity, and dependence on oral corticosteroids were observed in those given dupilumab as well, compared to placebo.

In their assessment of the medication’s safety profile, the team concluded that the most commonly reported adverse events (AEs) in the treatment cohort had been joint pain, peripheral edema, back pain, hypertension, limb injury, vision blurring, asthma, constipation, conjunctivitis, upper respiratory tract infections, and insomnia, with occurrences among at least 3 subjects.

The drug’s safety and efficacy for those with bullous pemphigoid remains under investigation and these evaluations have not yet been reviewed by regulatory authorities. The drug has, in the present moment, been given regulatory approval in over 60 countries for such diseases as atopic dermatitis, prurigo nodularis, and chronic spontaneous urticaria. Dupilumab’s development is a collaborative effort between Sanofi and Regeneron.

References

1. Press Release: Dupixent sBLA accepted for FDA priority review for the targeted treatment of bullous pemphigoid. Sanofi - Aventis Groupe. February 18, 2025. https://www.globenewswire.com/news-release/2025/02/18/3027482/0/en/Press-Release-Dupixent-sBLA-accepted-for-FDA-priority-review-for-the-targeted-treatment-of-bullous-pemphigoid.html.

2. 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, 2024. https://www.sanofi.com/en/media-room/press-releases/2024/2024-09-11-05-00-00-2944237.