Multi-Million Dollar Gene Therapy ZEVASKYN™ Increases Health Care Cost in Rare Skin Disorder – Recessive Dystrophic Epidermolysis Bullosa

Will This Gene Therapy Break Payers’ Budgets?

Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe, inherited skin disorder that has no cure, and until recently, had no FDA-approved therapies for treatment. On April 29^th, Abeona Therapeutics announced the FDA approval of ZEVASKYN™, the first of its kind, to treat RDEB wounds with a single surgical application directly onto wounds. Patients with RDEB may have more than one sheet surgically applied to each wound, depending on wound size, and more than one wound treated with ZEVASKYN. Despite the life-changing nature of this new therapy, the cost for ZEVASKYN is $3.1 million for every 12 sheets produced. As individuals begin to move over to this new treatment, the financial impact on payers could be detrimental if not foreseen.

What is Recessive Dystrophic Epidermolysis Bullosa (RDEB)?

Dystrophic Epidermolysis Bullosa (DEB) is an inherited skin disorder characterized by skin blisters and potential impact to inner lining of some organs and body cavities. DEB is a subtype of epidermolysis bullosa (EB) and is caused by one or more mutations on COL7A1 gene. DEB affects approximately 3.3 out of every 1 million in the US, or about 5% of all EB cases.

ZEVASKYN has been approved to treat recessive DEB (RDEB), which is considered the more severe form of DEB. Patients with RDEB have chronic blisters, nail dystrophy, and open wounds with skin fragility. The skin of patients with this disease is sometimes compared to butterfly wings or tissue paper to illustrate the extreme fragility. About 95% of RDEP patients develop pseudosyndactyly (fusion of hands or feet) or “mitten deformity” due to repeated blistering and scarring. Patients are at high risk for severe infections and hospitalizations, often relying on laborious, supportive care that manages symptoms but does not offer a cure. Complications of DEB progression could include growth delay, squamous cell carcinoma,  renal failure, and could also lead to early mortality.

Cost and Health Care Utilization Considerations

ZEVASKYN is the first FDA-approved therapy indicated for the treatment of wounds in adult and pediatric patients with RDEB. The therapy is unique in its delivery system, in which a single, surgical sheet with the patient’s own viable, gene-modified cells with functional copies of the COL7A1 gene is applied to the wound. The result would be an expression of collagen 7 (C7) protein and wound healing. Zevaskyn is the only FDA-approved therapy for RDEB, specifically.

ZEVASKYN performed strongly in clinical trials, demonstrating meaningful increases to quality of life for patients with RDEB through durable wound healing and reduction of pain and infection. In terms of financial outcomes, it is still difficult to measure whether the total cost of care for patients treated with ZEVASKYN would be less than those not treated. The estimated average total annual costs for DEB in the US range from $31,836 to $73,000. The estimated projected lifetime cost for treatment of RDEB could be between $10 million and $20 million. However, patients with the severest forms of the disease and most likely candidates for the therapy can cost considerably more to treat.

ZEVASKYN will add to the list of high-cost therapies, with a cost of $3.1 million per treatment, with up to 12 sheets produced. Each sheet of ZEVASKYN is 41.25 cm² (5.5 cm x 7.5 cm) or about the size of a credit card.  There is an estimated 25 days to manufacture the sheets from patient’s cells. Unlike other treatments, due to the nature of RDEB, ZEVASKYN is applied once to each wound. Patients with RDEB may have multiple wounds treated and multiple sheets could be used depending on each wound size. Patients would need to stay inpatient for 5-10 days of monitoring post-surgical application. In clinical trials, one patient received additional wound treatment as soon as 6 months after initial ZEVASKYN application due to such great clinical response. Thus, each treatment estimated at $3.1 million could be a substantial cost to payers. Abeona Therapeutics is offering an undisclosed warranty program with a percentage back to payers if patients require an additional treatment for the same site. Treatment considerations and specific qualifications, including location of wound, confirmation of genetic testing, and finding qualified treatment centers, may slow uptake of ZEVASKYN. Abeona Therapeutics has announced the launch of ZEVASKYN Q3 2025 with treatment for potentially 10-15 patients in 2025.

While these new topical treatments offer hope for patients and caregivers, there is not a single therapy available on the market that addresses the underlying genetic mutation. However, the reduction of chronic wound progression would reduce complications that may lead to early mortality in RDEB patients, especially infection, sepsis, and the development of squamous cell carcinoma.

What Can Payers Do?

At PSG, we’ve developed an extension of our integrated data analytics tool, Artemetrx, that leverages comprehensive healthcare data to help payers determine the risk within their own population of receiving a CGT within a given time period, looking as far out as 24 months. The extension, GeneCQ, now includes ZEVASKYN and its estimated impact to payers based on their own patient population. When deciding coverage and anticipating gene therapy risk, there are many considerations from all stakeholders in the healthcare ecosystem that could impact utilization, including impacted treatment population. Using integrated data to understand the risks and patient population to qualify for these high-cost therapies could help all payers understand the true landscape and impact of personalized medicine. The ability to anticipate high-cost gene therapies within your own given population could help with financial modeling, budget planning, and premium estimations for the upcoming year.

Although there is a place for these high-cost therapies, payers need to balance the cost, limited healthcare resource allocations, and access to appropriate healthcare expenditures. To prepare payers for high-cost claims, PSG recommends looking at all levels of coverage and cost containment strategies:

Appropriate utilization management (UM)

• UM programs, although often criticized, could help determine if the right patients qualify for high-cost gene therapies, based on current disease progression, other therapies available, and clinical monitoring parameters.

Proactive network and reimbursement strategies

• Considerations for network reimbursements for high-cost claimants, develop a process in which high-cost therapies are billed, and/or create a standard network for gene therapies where partnership with known centers of excellence can provide the best overall care.

Evaluation of care management programs

• Use of care management/overall patient care services to facilitate and ease patient care models and navigate different, unique steps for gene therapy.

Understanding what reimbursement strategies are available for each therapy

• Innovative warranty or value-based outcomes are available depending on treatment and disease monitoring parameters.

Monitoring high-cost claimants

• Establishing a claims monitoring process to identify outliers or to audit network contracts would help protect payers from unexpected, high-cost reimbursements.

As CGTs start to become more prevalent, they also increase the budgetary risk that payers face. This balance between prudent fiduciary practices and optimal member outcomes can certainly be a challenge, but there are proven strategies to help address it. If you would like to learn more about the potential upcoming CGTs in your pharmacy or medical benefit strategy, reach out to our expert team.

References:

• The Dystrophic Epidermolysis Bullosa Research Association (debra) of America. www.debra.org
• Zevaskyn Prescribing Information. Abeona Therapeutics, Inc. April 2025.
• U.S. FDA Approves Zevaskyn™ (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Reccessive Dystrophic Epidermolysis Bullosa (RDEB). Abeona Therapeutics. Press Release April 29, 2025
• Vyjuvek Prescribing Information. Krystal Biotech, Inc. May 2023
• Filsuvez Prescribing Information. Chiesi USA, Inc. May 2024.
• Bodke, A; Knott, C, et al. Economic Burden and Cost Drivers of Dystrophic Epidermolysis Bulosa: A Systemic Literature Review. Presentation at Nexus Values: London, United Kingdom. Access: https://www.ispor.org/docs/default-source/euro2023/isporeurope23bodkeee395poster133525-pdf.pdf?sfvrsn=e9d371e0_0. Accessed May5, 2025.
• Raymakers, PhD. Adam J. N.; Kesselheim, MD, JD, MPH, Aaron S.; et al. Estimated Spending on Beremagene Geperpavec for Dystrophic Epidermolysis Bullosa. JAMA Dermatol. 2024;160(3):297-302. doi:10.1001/jamadermatol.2023.5857

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