FDA

Dupixent approved for bullous pemphigoid

June 24, 2025

FDA approved Dupixent (dupilumab) for the treatment of adult patients with bullous pemphigoid (BP).

Efficacy

Approval was supported by results from the phase 2/3 LIBERTY-BP ADEPT trial (NCT04206553) involving 106 adults with a Bullous Pemphigoid Disease Area Index (BPDAI) activity score ≥24 on a scale of 0-360 and a weekly average Peak Pruritus NRS score ≥4 on a scale of 0-10. Participants were randomized to receive either SC Dupixent 600 mg (two 300 mg injections) on day 1, followed by 300 mg q2wks, or matching placebo for 52 weeks. All subjects were also initiated on a standard regimen of oral corticosteroids (OCS; prednisone or prednisolone, 0.5 or 0.75 mg/kg/day).

The primary endpoint was the proportion of subjects achieving sustained remission at week 36. Sustained remission was defined as the achievement of complete remission and off OCS no later than week 16, absence of disease relapse from the time of completion of the corticosteroid taper to week 36, and absence of rescue therapy during the 36-week double-blind treatment period. Secondary endpoints included total cumulative dose of OCS and proportion of subjects with a reduction in itch defined as a ≥4-point improvement (reduction) in the Peak Pruritus NRS.

A greater proportion of patients in the Dupixent group experienced sustained disease remission at 36 weeks vs. the placebo group (18.3% vs. 6.1%, respectively; 12.2% difference; 95% confidence interval [CI], -0.8 to 26.1). The proportion of patients with improvement of ≥4 points in Peak Pruritus NRS from baseline was 38.3% in the Dupixent arm vs. 10.5% in the placebo arm (difference, 27.8%; 95% CI, 11.6-43.4).

The proportion of participants who received rescue therapy during the 36-week treatment period was 53% in the Dupixent group and 79% in the placebo group. The median cumulative dose of OCS at week 36 was 2.8 g in the Dupixent group vs. 4.1 g in the placebo group.

Safety

The most common adverse reactions (≥2%) reported in the BP trial were arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis.

Sources:

Dupixent (dupilumab) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761055s070lbl.pdf Revised June 2025. Accessed June 23, 2025.

Dupixent® (dupilumab) approved in the US as the only targeted medicine to treat patients with bullous pemphigoid (BP). [News release]. 2025. https://newsroom.regeneron.com/news-releases/news-release-details/dupixentr-dupilumab-approved-us-only-targeted-medicine-treat