From Blisters to Bliss: A New Horizon in Bullous Pemphigoid Treatment

Select treatment options for bullous pemphigoid (BP) based on current guideline recommendations 

Discuss the rationale for using targeted therapies to optimize outcomes in patients with BP 

From Blisters to Bliss: A New Horizon in Bullous Pemphigoid Treatment: An Online Activity

Bullous Diseases

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<mux-player playback-id="Eczuk75wqXJMiGHAgpo3ZY2G01Gcbu8LOB00Dmnd49u9Q" metadata-viewer-user-id="Placeholder (optional)"></mux-player></div>James Del Rosso, DO, highlights the significance of the FDA approval of Dupixent for bullous pemphigoid, including clinical trial outcomes, safety profile, and implications for dermatology practiceSanofi and Regeneron have announced the FDA approval of Dupixent (dupilumab) for the treatment of adults with <a href="/conditions/bullous-diseases" target="_blank" rel="noopener noreferrer">bullous pemphigoid</a> (BP), a rare, relapsing chronic skin disease driven in part by type 2 inflammation. This marks the first FDA-approved treatment for BP, offering a new therapeutic pathway for a condition previously managed with broad immunosuppressants.Clinical trial insightsThe approval is based on results from the pivotal Phase 2/3 ADEPT trial, which evaluated Dupixent in adults with moderate-to-severe BP. Patients received Dupixent or placebo alongside a standard-of-care oral corticosteroid (OCS) tapering regimen.Key results at Week 36 include:<ul><li>Patients receiving Dupixent achieved sustained disease remission at a rate of 18.3%, compared to 6.1% in the placebo group, meeting the study’s primary endpoint</li><li>Clinically meaningful itch reduction was observed in 38.3% of patients treated with Dupixent, compared to 10.5% of those receiving placebo</li><li>Medians for cumulative OCS dose were lower in the Dupixent group (2.8 g) than placebo (4.1 g)</li></ul>Sustained disease remission was defined as complete clinical remission with successful OCS taper by Week 16 and no relapse or need for rescue therapy through Week 36.Safety profile&nbsp;The safety profile of Dupixent for BP was consistent with prior experience across other indications. Adverse events (≥2%) more commonly reported with Dupixent included arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis. One case of acute generalized exanthematous pustulosis was observed in the Dupixent group.Expanding therapeutic indications&nbsp; Dupixent is now approved by the FDA for the treatment of 8 distinct diseases that share an underlying mechanism of type 2 inflammation. These conditions span multiple organ systems, including the skin, lungs, and gastrointestinal tract. In addition to bullous pemphigoid, approved indications include <a href="/conditions/atopic-dermatitis" target="_blank" rel="noopener noreferrer">atopic dermatitis</a>, asthma, <a href="/conditions/chronic-spontaneous-urticaria" target="_blank" rel="noopener noreferrer">chronic spontaneous urticaria</a>, and <a href="/conditions/prurigo-nodularis" target="_blank" rel="noopener noreferrer">prurigo nodularis</a>. The addition of bullous pemphigoid expands the utility of Dupixent into a new category of autoimmune blistering diseases, offering a targeted treatment approach where limited options have historically been available.

Dupixent is the first and only FDA-approved treatment for bullous pemphigoid, demonstrating significant itch and blister reduction in clinical trials

Launch Alert: FDA Approves Dupixent (Dupilumab) for Adults with Bullous Pemphigoid

WEDNESDAY, June 25, 2025 (HealthDay News) -- For patients with recessive dystrophic epidermolysis <a href="/conditions/bullous-diseases" target="_blank" rel="noopener noreferrer">bullosa</a> (RDEB), a one-time surgical application of prademagene zamikeracel, an autologous COL7A1 gene-modified cellular sheet that is sutured onto a large, chronic RDEB wound, improves wound healing and pain versus standard of care, according to a study published online June 23 in The Lancet.Jean Y. Tang, M.D., Ph.D., from the Stanford University School of Medicine in California, and colleagues conducted a randomized open-label, phase 3 trial conducted at two institutions to examine the efficacy and safety of a one-time surgical application of prademagene zamikeracel in wound healing. Eligible patients were aged 6 years or older, with a confirmed clinical and genetic diagnosis of RDEB, and at least two chronic wounds. Large chronic wounds on participants were matched in pairs and randomly assigned to treatment with prademagene zamikeracel or control (standard of care).Eleven patients were enrolled, with 43 randomized wound pairs. The researchers found that at week 24, 81 and 16 percent of the treated and control wounds, respectively, were at least 50 percent healed from baseline (mean difference, 67 percent). From baseline to week 24, the mean change in wound pain was −3.07 and −0.90 in those receiving prademagene zamikeracel and controls, respectively (mean pairwise difference, −2.23). There were no serious treatment-related adverse events reported."With our novel gene therapy technique, we successfully treated the hardest-to-heal wounds, which were usually also the most painful ones for these patients," Tang said in a statement.Several authors disclosed ties to biopharmaceutical companies, including Abeona Therapeutics, which funded the study. The patent for prademagene zamikeracel is licensed to Abeona.<a target="_blank" rel="noopener noreferrer" href="https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)00778-0/abstract">Abstract/Full Text</a><a target="_blank" rel="noopener noreferrer" href="https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(25)01154-7/abstract">Editorial</a>

One-time surgical application of prademagene zamikeracel improves wound healing and pain

Gene Therapy Effective in Patients With Recessive Dystrophic Epidermolysis Bullosa

TUESDAY, June 24, 2025 (HealthDay News) -- The U.S. Food and Drug Administration has approved Dupixent (dupilumab) for the treatment of adult patients with <a href="/conditions/bullous-diseases" target="_blank" rel="noopener noreferrer">bullous pemphigoid</a>.The approval is based on results from the pivotal ADEPT phase 2/3 study. Patients with moderate-to-severe bullous pemphigoid were randomly assigned to receive Dupixent 300 mg (53 individuals) or placebo (53 individuals) added to standard-of-care oral corticosteroids. At 36 weeks, 18.3 percent of patients treated with Dupixent experienced sustained disease remission versus 6.1 percent with placebo. Additionally, 38.3 percent of patients treated with Dupixent achieved clinically meaningful itch reduction versus 10.5 percent with placebo. Finally, median cumulative oral corticosteroid dose was lower with Dupixent versus placebo (2.8 versus 4.1 grams).The most common adverse events (≥2 percent) observed in patients treated with Dupixent included arthralgia, conjunctivitis, blurred vision, herpes viral infections, and keratitis. One patient treated with Dupixent reported acute generalized exanthematous pustulosis."Until now, treating bullous pemphigoid was very challenging for elderly patients struggling with the debilitating impact of blisters and lesions, and potentially comorbid conditions," Alyssa Johnsen, M.D., Ph.D., global therapeutic area head for immunology and oncology development at Sanofi, said in a statement. "By addressing two central drivers of the underlying type 2 inflammation that contributes to bullous pemphigoid, Dupixent is the first targeted medicine to allow patients the potential to achieve sustained remission and reduce itch."Approval of Dupixent for this indication was granted to Sanofi and Regeneron.<a target="_blank" rel="noopener noreferrer" href="https://www.sanofi.com/en/media-room/press-releases/2025/2025-06-20-05-00-00-3102518">More Information</a>

Benefits include sustained disease remission, reductions in itch, lower oral corticosteroids dose

FDA Approves Dupixent for Bullous Pemphigoid

TUESDAY, April 29, 2025 (HealthDay News) -- The U.S. Food and Drug Administration has approved Zevaskyn (prademagene zamikeracel) for patients with the rare, genetic skin disorder recessive dystrophic epidermolysis bullosa.Zevaskyn is the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa, a skin condition characterized by extremely fragile skin that blisters and tears easily.FDA approval of Zevaskyn was based on findings from two clinical studies. In a single-center, open-label, phase 1/2a study involving 38 chronic wounds in seven patients, a single surgical application of Zevaskyn demonstrated long-term improvement at the treated sites during a median seven-year follow-up period.In the multicenter, randomized, intrapatient-controlled, phase 3 VIITAL study, evaluating the effectiveness of a single application of Zevaskyn on 43 large, chronic wounds, 81 percent of wounds demonstrated 50 percent or greater healing after six months. This result was statistically significant when compared with a matched control group of 43 wounds treated with the standard of care, where only 16 percent achieved the same level of healing.Across both studies, Zevaskyn was well tolerated, and no treatment-related serious adverse events were reported. The most common nonserious adverse events reported were procedural pain and itch, which only appeared to affect about 5 percent of patients."Based on the strength of our data across clinical trials, we are confident in Zevaskyn's ability to deliver long-term results after a single treatment application," Madhav Vasanthavada, Ph.D., chief commercial officer at Abeona Therapeutics, said in a statement. "We are committed to working closely with both commercial and government payers on outcome-based agreements that stand behind the promise of Zevaskyn for patients, and expedite access."Approval of Zevaskyn was granted to Abeona Therapeutics.<a target="_blank" rel="noopener noreferrer" href="https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel">More Information</a>

Zevaskyn is the first and only autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa

FDA Approves Zevaskyn for Rare, Genetic Skin Disorder

WEDNESDAY, Aug. 2, 2023 &nbsp;-- For patients with bullous pemphigoid (BP), dupilumab is associated with improved clinical symptoms, according to a study published online Aug. 2 in JAMA Dermatology.Liuqi Zhao, M.D., from Peking University First Hospital in China, and colleagues examined the efficacy and safety of dupilumab in patients with BP in a retrospective cohort study. A total of 146 adult patients with a clinical presentation of BP combined with immunological or pathological evidence, who received 300 mg of dupilumab every two weeks following an initial dose of 600 mg, were included in the study.The researchers found that 87.0 percent of the patients achieved disease control within four weeks (median time, 14 days). Overall, 35.6 and 8.9 percent of patients achieved complete remission and relapsed during the observation period, respectively. At week 64, the complete remission rate and cumulative relapse rate were 62.5 and 30.9 percent, respectively. After dupilumab treatment, rapid and sustained improvement in clinical indicators and laboratory examination results were seen, including Bullous Pemphigoid Disease Area Index scores, itching numerical rating scale scores, serum anti-BP180 and anti-BP230 antibodies, total immunoglobulin E levels, and eosinophil count. Overall, 73.3 percent of the patients did not report any adverse events. Infections and eosinophilia were the most common adverse events. Serum anti-BP180 antibody levels &gt;50 relative units/mL were associated with four-week disease control (odds ratio, 3.63; 95 percent confidence level, 0.97 to 12.61; P = 0.045), while relapse was more likely among male patients (hazard ratio, 10.97; 95 percent confidence level, 1.42 to 84.92; P = 0.02)."We found that dupilumab effectively alleviated clinical and serological indicators of patients with BP," the authors write. "The safety profile was favorable, while the risk of infection and eosinophilia were notable concerns."

Rapid and sustained improvement in clinical indicators and laboratory examination results seen after treatment

 Do Clinical Symptoms Improve With Dupilumab in Bullous Pemphigoid?

In episode 25 of Cutaneous Miscellaneous, our host, Nicholas Brownstone, MD, chats with Charlie Dunn, MD, board-certified pediatrician and Chief Dermatology Resident at KCU-GME Consortium / ADCS Orlando Dermatology Residency Program in Orlando, FL.&nbsp;

In episode 25 of Cutaneous Miscellaneous, our host, Nicholas Brownstone, MD, chats with Charlie Dunn, MD, board-certified pediatrician and Chief Dermatology Resident at KCU-GME Consortium / ADCS Orlando Dermatology Residency Program in Orlando, FL.&nbsp;The episode begins with board review on epidermolysis bullosa (EB). Dr Brownstone briefly highlights board review pearls on transport media used in dermatopathology. Next, Dr Dunn and Dr Brownstone discuss high-yield board review facts on the genetics, onset, diagnosis, morphology, associated clinical features, and prognosis of all the major subtypes of EB simplex, junctional EB and dystrophic EB. &nbsp;Dr Dunn and Dr Brownstone then discuss everything Camp Discovery. Dr Brownstone asks Dr Dunn about the history of Camp Discovery and which types of patients are eligible. Dr Dunn discusses a typical day at Camp Discovery including which activities are available and the kinds of counselors who staff the camp. Finally, Dr Dunn provides detailed information on how to refer a pediatric patient to the camp, which is completely free for the camper and their family! &nbsp;Year after year, there are open spots at Camp Discovery. Listen now to learn about this amazing experience for your pediatric patients with skin disease and how you can make a referral!More InformationGeneral information on <a target="_blank" rel="noopener noreferrer" href="https://www.aad.org/public/public-health/camp-discovery">Camp Discovery</a> from the AADApply to be a <a target="_blank" rel="noopener noreferrer" href="https://www.aad.org/public/public-health/camp-discovery/volunteer">Camp Discovery Volunteer</a>Access the <a target="_blank" rel="noopener noreferrer" href="https://www.aad.org/public/public-health/camp-discovery/referrals">Camp Discovery Patient Referral Form</a>For questions or more detailed information, please contact Janine Mueller at <a href="mailto:jmueller@aad.org" target="_blank" rel="noopener noreferrer">jmueller@aad.org</a> or call (847) 240-1737.&nbsp;Cutaneous Miscellaneous Boards Booster!In this new feature, we’ll put your knowledge to the test with high-yield board review questions written by dermatology residents and fellows and sourced from key resources like Bolognia’s Dermatology, Andrews’ Dermatology, and Alikhan’s Review of Dermatology. These quiz questions will test your skills on the board review topic that’s discussed in each episode. <a target="_blank" rel="noopener noreferrer" href="https://www.research.net/r/FGZ6JV7?Email=[Email_value]">Click here to begin.</a>

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Launch Alert: FDA Approves Dupixent (Dupilumab) for Adults with Bullous Pemphigoid

Gene Therapy Effective in Patients With Recessive Dystrophic Epidermolysis Bullosa

FDA Approves Dupixent for Bullous Pemphigoid