Dupixent Receives Priority Review for Bullous Pemphigoid with PDUFA Date Set for June 2025

On February 18, Sanofi and Regeneron announced that the FDA has accepted the supplemental biologics license application (sBLA) for Dupixent (dupilumab) to treat Bullous Pemphigoid (BP) in adults, granting it priority review status. The PDUFA date is set for June 20, 2025. If approved, Dupixent will be the first and only targeted therapy for BP in the United States.

BP is a chronic, debilitating, and recurrent skin condition characterized by severe itching, blisters, redness, and pain. It predominantly affects older adults and can lead to complications such as skin bleeding and crusting, which increase the risk of infections and impact daily functioning.

In September 2024, promising results emerged from the Phase II/III ADEPT trial for Dupixent in treating BP. This 52-week randomized, double-blind, placebo-controlled study involved 106 patients and assessed the efficacy and safety of Dupixent (300 mg every two weeks) in combination with oral corticosteroids (OCS) compared to placebo plus OCS for adults with moderate to severe BP. The main endpoint was the proportion of patients achieving sustained disease remission by week 36.

Initially, patients received either Dupixent or placebo along with OCS. After achieving disease control for two consecutive weeks, patients began a tapering process for OCS, which was completed before week 16. Subsequently, patients continued on Dupixent or placebo, unless rescue treatment was required, for at least 20 weeks.

Results at week 36 showed that a higher percentage of patients in the Dupixent group achieved sustained remission compared to the placebo group (20% vs. 4%, p=0.0114). Additionally, Dupixent-treated patients had a higher rate of disease stability after OCS tapering (59% vs. 16%, nominal p=0.0023) and a lower need for rescue treatment (42% vs. 12%, nominal p=0.0004). Although the percentage of patients achieving complete remission and discontinuing OCS by week 16 was higher in the Dupixent group, it did not reach statistical significance (38% vs. 27%).

The study also met all secondary endpoints, including a greater reduction in disease severity (77% vs. 51%, p=0.0021) and a higher percentage of patients experiencing at least a 90% reduction (41% vs. 10%, p=0.0003). Moreover, the Dupixent group reported a greater reduction in itching (52% vs. 27%, p=0.0021) and a significant decrease in OCS usage days (40 vs. 13 days, p=0.0072).

In terms of safety, the overall incidence of adverse events (AEs) was 96% for both groups. However, the Dupixent group reported more frequent AEs such as peripheral edema, joint pain, back pain, blurred vision, hypertension, asthma, conjunctivitis, constipation, upper respiratory infections, limb injuries, and insomnia. Notably, there were fatal AEs in the Dupixent group, while the placebo group had two deaths due to AEs.

This potential advancement in treating BP with Dupixent offers hope for patients suffering from this challenging condition.