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

Regeneron Pharmaceuticals, Inc. and Sanofi have announced that their medication Dupixent (dupilumab) has received approval from the U.S. Food and Drug Administration (FDA) for the treatment of adults with bullous pemphigoid (BP), a rare and chronic skin condition. This approval marks a significant advancement in therapeutic options for BP, a condition that predominantly affects the elderly and can lead to debilitating symptoms like severe itching and painful blisters.

Bullous pemphigoid is a serious skin disease affecting approximately 27,000 adults in the United States. Despite its rarity, the disease poses significant challenges in terms of management, especially since current treatments primarily involve systemic corticosteroids, which can have undesirable side effects. Dupixent's approval offers a new treatment avenue, directly targeting the underlying type 2 inflammation associated with the disease, potentially reducing the need for corticosteroids.

The FDA's decision to approve Dupixent for BP is based on the results from the pivotal ADEPT Phase 2/3 trial. This study evaluated the effectiveness and safety of Dupixent in comparison to a placebo in adults with moderate-to-severe BP. Participants in the trial were administered Dupixent along with standard-of-care oral corticosteroids, with outcomes showing significant improvements in sustained disease remission, reduced itch, and decreased reliance on corticosteroids.

Patrick Dunn, Executive Director at the International Pemphigus and Pemphigoid Foundation, emphasized the impact of Dupixent's approval. He noted that patients with BP have historically had limited treatment options, and Dupixent introduces a novel approach that could alleviate the severe symptoms associated with the disease. This sentiment was echoed by George D. Yancopoulos, a leading researcher at Regeneron, who highlighted the broader potential of Dupixent to transform treatment paradigms for various conditions driven by type 2 inflammation.

Dupixent has already been approved for multiple conditions characterized by type 2 inflammation, including asthma and atopic dermatitis. Its safety profile has been well-documented across a wide range of patients, from infants to the elderly, making it a versatile treatment option for various inflammatory diseases.

The pivotal trial evaluating Dupixent's efficacy in BP involved 106 adults over a 52-week treatment period. Patients received Dupixent or a placebo in combination with oral corticosteroids. The study's primary endpoint focused on the proportion of patients achieving sustained disease remission at 36 weeks. The results were promising, indicating Dupixent's potential to significantly improve disease outcomes for BP patients.

Dr. Alyssa Johnsen, representing Sanofi, underscored the importance of this approval for BP patients, many of whom struggle with the disease's debilitating effects and co-morbid conditions. Dupixent's ability to address key drivers of type 2 inflammation marks it as the first targeted treatment offering the potential for sustained remission and itch reduction in BP.

The FDA had previously granted Dupixent Orphan Drug Designation for BP due to the rarity of the condition. This designation is for treatments intended for diseases affecting fewer than 200,000 people in the U.S. The approval also underwent a Priority Review, underscoring the medication's potential as a significant advancement in the treatment of serious conditions.

Overall, the approval of Dupixent for bullous pemphigoid represents a promising development for patients and healthcare providers. As further applications are under review worldwide, including in the EU, Japan, and China, Dupixent could become an essential component in managing this challenging and rare skin disease.