An article from Medscape highlighted recent advances in treatment that are reshaping the clinical approach to epidermolysis bullosa (EB), transitioning care from largely supportive management to therapies that address genetic defects and accelerate wound repair. Speaking at a recent pediatric dermatology meeting, Emily Gorrell, DO, highlighted how multiple newly approved options are changing outcomes for patients (especially children) with this rare and often devastating condition.
From Symptom Control to Targeted Treatment
Until recently, EB management focused on minimizing symptoms such as pain, itching, infection, and chronic wounds, as well as monitoring for complications like aggressive squamous cell carcinoma. However, since 2022, the therapeutic landscape has broadened significantly. Clinicians now have access to treatments that not only improve wound healing but also target the underlying molecular drivers of disease.
This shift requires dermatologists to carefully consider treatment selection and application strategies to optimize outcomes in different EB subtypes.
Gene Therapy Approaches Gain Ground
Two of the most significant advances involve gene delivery therapies designed to restore functional type VII collagen (COL7A1) in dystrophic EB.
Beremagene geperpavec (B-VEC, Vyjuvek), approved in 2023 for both recessive and dominant dystrophic EB, delivers corrective genetic material directly to wounds using a viral vector. A 2025 label expansion allows its use in newborns and enables at-home caregiver application, increasing accessibility.
Effective use of B-VEC requires meticulous wound preparation. Clinicians recommend treating one wound at a time, starting with the most severe. Certain topical agents—such as antiseptics or oxidizing products—should be avoided prior to application, as they may reduce efficacy. The therapy is applied in a grid-like pattern and covered with specialized dressings to ensure proper absorption.
Prademagene zamikeracel (pz-cel; Zevaskyn), approved in 2025 for recessive dystrophic EB, uses autologous keratinocyte sheets genetically corrected in a manufacturing facility. After a skin biopsy, grafts are produced over several weeks and surgically applied to wounds in specialized centers.
To maximize success, patients should be optimized before the procedure through infection control, wound care, and nutritional support. Post-application recovery includes strict bed rest, antibiotic therapy, and pain management to promote graft integration.
Topical Therapy Expands Options
In addition to gene-based therapies, topical treatments are playing an increasingly important role. A birch bark–derived triterpene gel (Filsuvez) received FDA approval in 2023 for patients aged 6 months and older with junctional or dystrophic EB.
The gel can be applied at each dressing change, either directly to wounds or via dressings. Clinicians emphasize appropriate usage—overapplication or use on unaffected skin may lead to residue buildup. Because the formulation lacks preservatives, each tube must be used immediately after opening.
Although approved for specific EB subtypes, off-label use has expanded to EB simplex, and the product is frequently combined with other therapies. Experts stress that these newer treatments are typically used in combination rather than as standalone interventions.
Access, Cost, and Genetic Confirmation
Despite therapeutic progress, significant barriers remain. Costs vary widely, from approximately $1,700 per tube for topical therapy to over $3 million for a full course of cell-based treatment. Insurance access has historically been challenging.
Genetic testing has become a critical step in accessing advanced therapies, as confirmation of EB subtype is often required for coverage. Programs such as Decode DEB offer no-cost genetic testing, helping facilitate diagnosis without sharing patient data with sponsors.
Ongoing Challenges and Future Directions
Experts caution that while these therapies improve skin-related manifestations, they do not fully address systemic or extracutaneous complications of EB, such as mucosal involvement. In addition, treatment durability may vary—for example, the effects of topical gene therapy may diminish over time, requiring repeat application.
Nevertheless, the outlook for patients with EB is markedly improved compared to previous years. Emerging therapies, including biologics aimed at reducing severe itch and investigational treatments in clinical trials, continue to broaden the pipeline.
As Lawrence Eichenfield, MD, noted, EB remains one of the most complex conditions in pediatric dermatology. However, the rapid expansion of therapeutic options—spanning approved, off-label, and investigational approaches—represents a significant step forward in improving quality of life for patients and their families.
Takeaway: The management of epidermolysis bullosa is undergoing a fundamental transformation, with gene therapies, engineered skin grafts, and novel topical agents offering new hope. While challenges remain, particularly regarding cost and systemic disease manifestations, the field is entering a period of unprecedented therapeutic progress.
