Press Release: Dupixent sBLA accepted for FDA priority review for the targeted treatment of bullous pemphigoid
Dupixent sBLA accepted for FDA priority review for the targeted treatment of bullous pemphigoid
- If approved, Dupixent would be the first and only targeted medicine to treat BP in the US; FDA decision expected by June 20, 2025
- Priority review granted based on positive pivotal results demonstrating significant improvements in sustained disease remission with Dupixent compared to placebo
- BP is a chronic, debilitating and relapsing skin disease with underlying type 2 inflammation characterized by intense itch and blisters, reddening of the skin and painful lesions
Paris and Tarrytown, NY, February 18, 2025. The US Food and Drug Administration (FDA) has accepted for priority review the supplemental biologics license application (sBLA) for Dupixent (dupilumab) to treat adults with bullous pemphigoid (BP).
The sBLA is supported by data from a pivotal study evaluating the efficacy and safety of Dupixent in 106 adults with moderate-to-severe BP. The primary endpoint was met, with five times more Dupixent patients achieving sustained disease remission compared to those on placebo. Sustained disease remission was defined as complete clinical remission with completion of oral corticosteroids (OCS) taper by week 16 (off OCS treatment and only treated with Dupixent for at least 20 weeks) without relapse and no rescue therapy use during the 36-week treatment period. The study also showed that Dupixent significantly reduced disease severity, itch, and use of OCS compared to placebo.
Adverse events more commonly observed with Dupixent (in at least 3 patients) compared to placebo included peripheral edema, arthralgia, back pain, blurred vision, hypertension, asthma, conjunctivitis, constipation, upper respiratory tract infection, limb injury, and insomnia.
BP is a chronic, debilitating, and relapsing skin disease with underlying type-2 inflammation that typically occurs in an elderly population. It is characterized by intense itch and blisters, reddening of the skin, and painful lesions. The blisters and rash can form over much of the body and cause the skin to bleed and crust, resulting in patients being more prone to infection and affecting their daily functioning. Approximately 27,000 adults in the US live with BP that is uncontrolled by systemic corticosteroids.
Priority review is granted to regulatory applications seeking approval for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Dupixent was previously granted orphan drug designation by the FDA for BP, which applies to investigational medicines intended for the treatment of rare diseases that affect fewer than 200,000 people in the US.
The safety and efficacy of Dupixent in BP are currently under clinical assessment and have not been evaluated by any regulatory authority.
About Dupixent
Dupixent (dupilumab) is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL4) and interleukin-13 (IL13) pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type-2 inflammation in phase 3 studies, establishing 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.
Dupixent has received regulatory approvals in more than 60 countries in one or more indications including certain patients with atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous urticaria, and chronic obstructive pulmonary disease in different age populations. More than one million patients are being treated with Dupixent globally.
Dupilumab development program
Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical studies involving more than 10,000 patients with various chronic diseases driven in part by type-2 inflammation.
In addition to the currently approved indications, Sanofi and Regeneron are studying dupilumab in a broad range of diseases driven by type-2 inflammation or other allergic processes in phase 3 studies, including chronic pruritus of unknown origin, bullous pemphigoid, and lichen simplex chronicus. These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority.
About Regeneron
Regeneron (NASDAQ:REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.
Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.
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About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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