FDA Approves Dupixent for Rare Skin Disease Bullous Pemphigoid

The U.S. Food and Drug Administration (FDA) has approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid (BP). This approval marks the first targeted therapy for the condition, based on pivotal study results demonstrating improvements in sustained disease remission, itch reduction, and decreased oral corticosteroid use compared to placebo.

Bullous pemphigoid is a chronic, relapsing, and rare skin disease characterized by intense itch, painful blisters, and lesions, primarily affecting elderly individuals. Approximately 27,000 adults in the U.S. with uncontrolled BP have limited treatment options, which can suppress the immune system and add to the disease burden.

The FDA's decision was based on data from the pivotal Phase 2/3 ADEPT study. At 36 weeks, the study showed that 18.3% of patients treated with Dupixent achieved sustained disease remission, compared to 6.1% in the placebo group. Additionally, 38.3% of Dupixent-treated patients experienced a clinically meaningful reduction in itch.

With this approval, Dupixent is now indicated in the U.S. for eight distinct diseases driven by underlying type 2 inflammation, spanning skin, gut, and respiratory conditions in patients from infants to the elderly. Sanofi is pursuing regulatory reviews for Dupixent in BP in other regions, including Europe, Japan, and China.