Sanofi’s sBLA for Dupixent Gets Priority Review for Bullous Pemphigoid Indication

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Sanofi announced that FDA has accepted its supplemental biologics license application (sBLA) for dupilumab (brand name Dupixent), a fully human monoclonal antibody (mAb), and granted the application priority review status. The company is seeking an approval for the targeted treatment of adults with bullous pemphigoid (BP), which is a chronic, debilitating, and relapsing skin disease with underlying type 2 inflammation. It is characterized by intense itch and blisters, reddening of the skin, and painful lesions. If approved, dupilumab would become the first and only targeted medicine for this indication, according to Sanofi in a Feb. 18, 2025 press release. A decision is expected from FDA by June 20, 2025 (1).

The priority review was granted based on positive results from a pivotal trial that showed significant improvements in sustained remission of BP in patients using dupilumab compared to placebo. The study evaluated the efficacy and safety of the mAb in 106 adults with moderate-to-severe BP. The study met its primary endpoint, with five times more patients on dupilumab treatment achieving sustained remission compared to those on placebo. The study also showed that treatment with dupilumab significantly reduced disease severity, itch, and use of oral corticosteroids compared to placebo.

Blisters and rash resulting from BP can form over much of the body and cause bleeding and crusting of the skin, which in turn makes patients more susceptible to infection and affects daily functioning. Approximately 27,000 adults in the United States are affected by BP, which cannot be controlled with systemic corticosteroids (1).

In addition to the priority review status, dupilumab (under the Dupixent brand name) was previously granted orphan drug designation by FDA for this indication. According to Sanofi in the release, “[t]he safety and efficacy of Dupixent in BP are currently under clinical assessment and have not been evaluated by any regulatory authority” (1).

Dupilumab works by inhibiting the signaling of the interleukin-4 (IL4) and interleukin-13 (IL13) pathways. It is not an immunosuppressant. The mAb’s development program has shown in Phase III studies that it offers significant clinical benefit and a decrease in type 2 inflammation. These results determine that IL4 and IL13 are “two of the key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases,” according to the press release.

Dupilumab has received regulatory approvals in more than 60 countries under the brand name Dupixent. Indications for which it is approved include atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous urticaria, and chronic obstructive pulmonary disease in different age populations.

Dupilumab is being jointly developed under a global collaboration between Sanofi and Regeneron Pharmaceuticals (Regeneron). The companies formed the collaboration in November 2007 under which they are using Regeneron's proprietary VelociSuite of technologies to discover, develop, and commercialize fully human therapeutic antibodies (2). Dupilumab has been studied across more than 60 clinical studies to date. The studies collectively involve more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation.

Beyond the currently approved indications, Sanofi and Regeneron are conducting Phase III studies in the use of dupilumab in a broad range of diseases caused by or resulting from type 2 inflammation or other allergic processes, including chronic pruritus of unknown origin, BP, and lichen simplex chronicus.

References

1. Sanofi. Dupixent sBLA Accepted for FDA Priority Review for the Targeted Treatment of Bullous Pemphigoid. Press Release. Feb. 18, 2025.
2. Regeneron Pharmaceuticals. Regeneron Initiates Major Global Collaboration with Sanofi-aventis to Develop and Commercialize Fully-Human Therapeutic Antibodies. Press Release. Nov. 29, 2007.