Dupixent® Approved in U.S. as Sole Targeted Bullous Pemphigoid Treatment

Regeneron Pharmaceuticals, Inc. and Sanofi have announced that the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for adult patients with bullous pemphigoid (BP). BP is a chronic and severe skin condition mostly affecting the elderly, characterized by intense itching and painful blisters. This condition is associated with type 2 inflammation and often leads to skin damage, increased infection risk, and significant impact on daily life. Treatment options for BP have been limited, frequently involving systemic corticosteroids which can further burden patients by affecting their immune systems.

Dupixent’s approval is based on pivotal clinical trial results demonstrating effectiveness in achieving sustained disease remission and reducing the need for oral corticosteroids, compared to placebo. The ADEPT Phase 2/3 trial, involving adults with moderate-to-severe BP, highlighted the drug’s potential benefits. Participants receiving Dupixent showed significant improvements in itch reduction and sustained remission. The study had participants undergo a protocol-defined corticosteroid tapering regimen if disease control was maintained. Results after 36 weeks demonstrated that 18.3% of those on Dupixent experienced sustained disease remission, a notable improvement over the 6.1% in the placebo group. Additionally, Dupixent recipients reported a higher reduction in itch severity and used a lower median cumulative dose of corticosteroids (2.8 grams) compared to placebo recipients (4.1 grams).

Common side effects observed in patients treated with Dupixent included joint pain, conjunctivitis, blurred vision, and herpes viral infections. There was also one case of acute generalized exanthematous pustulosis among Dupixent patients. The trial involved randomizing 106 adults to receive either Dupixent or placebo every two weeks, alongside oral corticosteroids, with a taper initiated upon achieving disease control. Patients continued on Dupixent or placebo post-taper, with rescue treatments allowed if necessary.

This FDA approval marks Dupixent’s expansion in treating various diseases driven by type 2 inflammation, with its portfolio now covering skin, gut, and respiratory conditions. Previously, Dupixent received Orphan Drug Designation for BP, a status for rare diseases affecting fewer than 200,000 people in the U.S. Sanofi and Regeneron are pursuing additional regulatory approvals globally, including in the EU, Japan, and China, to make Dupixent available to more patients.

The approval signifies a significant advancement for BP patients, who previously faced limited treatment choices. Patrick Dunn, Executive Director at the International Pemphigus and Pemphigoid Foundation, emphasized the relief this approval brings to patients and caregivers. Regeneron’s George D. Yancopoulos celebrated Dupixent’s potential to transform treatment paradigms for a variety of diseases linked to type 2 inflammation, highlighting its established safety profile across diverse demographic groups.

Dupixent, a fully human monoclonal antibody, functions by inhibiting interleukin-4 (IL-4) and interleukin-13 (IL-13) signaling, key drivers of type 2 inflammation. While not an immunosuppressant, Dupixent has demonstrated considerable clinical benefits in reducing type 2 inflammation across several chronic diseases. Invented using Regeneron’s VelocImmune® technology, Dupixent is administered subcutaneously and is intended for use under healthcare professional guidance.

Overall, the FDA’s approval of Dupixent for BP represents a significant step forward in providing a targeted treatment option for patients suffering from this debilitating condition, offering hope for improved management and quality of life.