Dupilumab is now the first and only FDA-approved targeted therapy for bullous pemphigoid, demonstrating sustained disease remission and reduced corticosteroid dependence in a pivotal trial.
The U.S. Food and Drug Administration (FDA) has approved dupilumab (Dupixent) as the first targeted therapy for adults with bullous pemphigoid (BP), a rare, chronic autoimmune skin disease characterized by pruritus, blistering, and widespread skin involvement. Affecting approximately 27,000 adults in the U.S., BP disproportionately impacts older patients and often requires prolonged use of systemic corticosteroids, which may increase morbidity in this population.
Approval was based on results from the ADEPT Phase 2/3 randomized, double-blind, placebo-controlled trial evaluating dupilumab 300 mg every 2 weeks versus placebo, both in combination with oral corticosteroids (OCS), in 106 adults with moderate-to-severe BP. The primary endpoint, sustained disease remission at 36 weeks, was achieved in 18.3% of patients receiving dupilumab compared with 6.1% receiving placebo (difference 12.2%). Clinically meaningful pruritus reduction was reported in 38.3% of the dupilumab group versus 10.5% of the placebo group. Median cumulative OCS dose was 2.8 g in the dupilumab arm versus 4.1 g in the placebo arm.
Adverse events more frequently reported in the dupilumab arm (≥2%) included arthralgia, conjunctivitis, herpes virus infections, blurred vision, and keratitis. One case of acute generalized exanthematous pustulosis occurred in a patient treated with dupilumab.
Dupilumab is a fully human monoclonal antibody that inhibits interleukin-4 and interleukin-13 signaling, key drivers of type 2 inflammation. It is not immunosuppressive and is administered via subcutaneous injection biweekly following an initial loading dose. In BP, treatment is initiated alongside a tapering course of OCS.
Dupilumab is now approved in the U.S. for eight type 2 inflammatory conditions spanning dermatologic, respiratory, and gastrointestinal indications. It was granted Priority Review and Orphan Drug Designation for BP and is currently under regulatory review in other global markets. Regeneron and Sanofi continue to evaluate dupilumab in other type 2 inflammation–related conditions, including chronic pruritus of unknown origin and lichen simplex chronicus.
Source: Regeneron
