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Denver — Thanks to treatments approved by the FDA in recent years, pediatric dermatologists’ strategy for epidermolysis bullosa (EB) has shifted from providing supportive care to correcting underlying causes and boosting wound healing, Emily Gorrell, DO, said at the annual Society for Pediatric Dermatology Pre-AAD meeting.

Before 2022, she said, management of EB’s cutaneous manifestations was limited to managing pain and itching, preventing and treating infections, supporting wound healing, and watching for squamous cell carcinomas, which are often aggressive in the context of EB. Today, dermatologists have several effective therapies from which to choose — and maximizing their use requires knowing where, how, and when to apply them, said Gorrell, assistant professor of dermatology at the University of Colorado Anschutz Medical Campus, Aurora, Colorado.

Addressing Genetic Defects

Both beremagene geperpavec (B-VEC, Vyjuvek) and prademagene zamikeracel (pz-cel; Zevaskyn) use viral vectors to deliver functional COL7A1 to affected skin. B-VEC earned FDA approval in 2023 for recessive dystrophic EB (RDEB) and dominant dystrophic EB (DEB). A label update approved by the FDA in September 2025 expanded the approval to include newborns and allowed application of the treatment at home by caregivers.

Gorrell summarized B-VEC application instructions as follows:

• Start with the most troublesome wound; treat until it heals before advancing to other wounds.
• Avoid products including hypochlorous acid, bleach baths, and vinegar for 24 hours before B-VEC application. Other topicals such as cannabidiol, chlorhexidine, and hydrogen peroxide also can hinder B-VEC efficacy.
• Remove ointments, scabs, and dead skin at the wound area.
• Place droplets 1 cm apart in a gridlike pattern without touching the syringe to the skin.
• Cover the wound with a hydrophobic dressing (so the drug absorbs into the wound, not the dressing) for at least 8 hours until dressing changes.
• Apply standard dressings such as petroleum gauze topped with Mepilex on top of the hydrophobic dressing.

Keratinocyte Sheets

Pz-cel earned FDA approval for RDEB in April 2025. While waiting 25 days for a manufacturing facility to produce up to 12 credit-card-sized sheets of genetically corrected keratinocytes from skin biopsies, Gorrell said, maximizing this treatment’s efficacy requires optimizing patients’ nutrition, wound care, and overall health. “Most importantly,” she said, “eradicate as much bacteria and infection as possible — give patients antibiotics. Make sure they’re bathing every day and using good cleansers and antiseptics.”

Treatment is administered at a qualified treatment center. Currently, there are six such centers, with Children’s Hospital of Philadelphia, Philadelphia, being the most recent addition in May.

When ready, the clear keratinocyte sheets are clipped to petrolatum-covered gauze backing, then sutured to selected wounds (after debridement and cauterization) in an operating room with patients under anesthesia. Keeping patients on hospital bed rest for 7 days post-procedure — during which time antibiotics and pain control are crucial — allows the grafts to take, Gorell said. Two to 3 weeks after hospital discharge, sutures dissolve, and the gauze backing falls off.

Birch Bark Triterpenes

A topical gel containing birch bark triterpenes (Filsuvez), formerly known as Oleogel-S10, earned FDA approval for patients 6 months or older with junctional or dystrophic EB in 2023, after the European Medicines Agency approval in 2022. It can be applied to all wounds at each dressing change, Gorrell said.

Patients can apply the gel directly to wounds, she added, or to a bandage (in roughly the shape of the wound). Applying the gel too thickly or to unaffected skin can create an unpleasant waxy residue, she cautioned. Additionally, patients can apply the medication directly, without dressings, to hard-to-dress areas such as the scalp and ears. Because the product contains no preservatives, Gorrell added, patients must use the full tube at once.

“Off-label,” she said, “we’ve been using Oleogel-S10 for EB simplex, which I’m very excited about. We’ve also been using it in combination with other therapies like B-VEC. None of these therapies are monotherapies. You’re going to be using everything together to try to help patients. Just don’t apply the Oleogel the same day you’re applying B-VEC.” Additional emerging uses for this treatment and for B-VEC include after hand surgery and in areas including the eye.

Genetic Testing Requirement

The new therapies are hardly inexpensive, Gorrell said, with costs ranging from $1700 per 23 g tube (Filsuvez, Oleogel-S10) to $3.1 million (pz-cel) per course. And historically, she added, getting insurance coverage for EB treatment was difficult.

Now, Gorrell said that if any type of EB is suspected, patients can get free genetic testing through the Decode DEB program. Genetic confirmation of EB diagnoses is necessary for access to newer therapies, she said. Although Krystal Biotech sponsors the program, said Gorrell, the company receives no patient data through it.

Overall, Lawrence Eichenfield, MD, told Medscape Dermatology, Gorrell’s presentation provided “an excellent summary and explanation of the many changes happening in the management of epidermolysis bullosa.” Eichenfield, professor of dermatology and pediatrics at the University of California, San Diego, and chief of pediatric and adolescent dermatology at Rady Children’s Hospital in San Diego, attended the presentation and provided commentary afterward.

“EB remains among the most challenging set of conditions we manage in pediatric dermatology,” he said. And the last few years have been exciting, he added, because dermatologists have a new group of therapies — whether approved for EB, off-label, or in clinical trials — that may prove “incredibly useful” in improving wound healing, decreasing pain, and improving the lives of patients with EB and their families.

Recently approved therapies are not without limitations, Eichenfield noted. None of them address the many extracutaneous and systemic manifestations of EB, which can include mucosal erosions that create difficulty swallowing, he explained.

Physicians who treat EB are aware of the high costs of pz-cel and of B-VEC (around $24,500 per vial), he added, and that wound healing with the latter may be temporary because the new type VII collagen can wear out over time. Likewise, Eichenfield said, pz-cel cannot treat mucosa or internal organs.

Still, Eichenfield said, the outlook for people with EB remains positive. As with approved therapies, he expressed excitement over off-label treatments, including those directed at minimizing the severe pruritus that affects many patients with EB. Examples include dupilumab and potentially other biologic or novel small-molecule agents, he said, adding: “the large list of active clinical trials in EB is also itself a miracle of hope for our patients and families.”

Gorrell reported being a consultant, advisory board member, and clinical investigator for Abeona Therapeutics; an advisor and investigator for Chiesi USA and Krystal Biotech; and an investigator for Castle Creek Biosciences, Incyte, Pfizer, Phoenicis (now Kamal Therapeutics), Phoenix Tissue Repair, RHEACELL, Sanofi, and TWi Biotechnology. Eichenfield reported serving as a consultant for Abeona, Chiesi, and Krystal Biotech.

John Jesitus is a Denver-based freelance medical writer and editor.