Obtain prior authorization (Vyjuvek and Zevaskyn)

Prior authorization is required before initiating beremagene geperpavec-svdt (Vyjuvek) or prademagene zamikeracel (Zevaskyn). Initial authorization for Vyjuvek is for 6 months; reauthorization is 12 months when continuation criteria are met. Zevaskyn prior approval limits are quantity 12 sheets and duration 6 months.

• Vyjuvek initial authorization period: 6 months (see product-specific criteria).
• Vyjuvek reauthorization period: 12 months if initial criteria and clinical response documented.
• Zevaskyn prior approval limits: Quantity 12 sheets; Duration 6 months.

Expect medical necessity review and eligibility checks

Some gene-therapy products and related services are subject to medical necessity review and prior authorization; coverage may vary by line of business and state/federal mandates.

• State or federal mandates (e.g., Federal Employee Program) may alter investigational determinations and require medical necessity review.
• This policy may not apply to certain lines of business (Federal Employee Program, Medicare Supplement, Medicare Advantage, Medicaid, certain self-insured groups).

Confirm genetic and diagnostic documentation in prior auth

Prior authorization must document genetically confirmed COL7A1 mutation(s) for Vyjuvek or biallelic COL7A1 mutations with NC1+ expression for Zevaskyn, along with matching clinical diagnosis of RDEB.

• Vyjuvek: documented COL7A1 mutation(s) and clinical manifestations consistent with dystrophic epidermolysis bullosa.
• Zevaskyn: documented biallelic pathogenic COL7A1 mutations and positive NC1 expression in skin.

Verify wound meets trial eligibility (size, duration, stage)

For prior authorization, confirm the treated wound(s) meet trial-derived eligibility: area ≥20 cm2, present for ≥6 months, and Stage 2 (open skin wound with partial-thickness loss of dermis).

• Wound area: ≥20 cm2.
• Wound duration: present for ≥6 months.
• Wound stage: Stage 2—partial-thickness loss of dermis not extending into subcutaneous tissue.

Assess for squamous cell carcinoma exclusion

The pivotal trials excluded patients with current or prior squamous cell carcinoma at the treatment site; evaluate targeted wound sites for any history or active SCC before approval.

• Document absence of active squamous cell carcinoma at targeted wound(s).
• Document no history of squamous cell carcinoma at the treatment site.

Include trial-aligned clinical documentation with PA

Prior authorization must include clinical documentation aligned with pivotal trial inclusion: diagnosis, genetic test results, wound assessments, and prior standard care; cite the RCTs used as evidence when applicable.

• Include diagnosis and genetic mutation status (COL7A1) where applicable.
• Provide wound assessments (size, duration, stage, location) and documentation of prior standard wound care.
• Reference clinical response endpoints when requesting reauthorization (e.g., complete wound closure).

Prior authorization implied for gene therapy codes

Prior authorization is required for these gene therapies and associated specialty products; submit benefit-plan–specific authorization using the policy’s listed codes to obtain approval before treatment.

• Relevant codes referenced in the policy (for prior auth submission) include J3401 (Vyjuvek) and J3389 (Zevaskyn), plus supporting CPT/ICD-10-PCS/ICD-10-CM codes.
• Obtain member-specific benefit authorization prior to treatment.

Prescriber attestation: no prior Zevaskyn treatment

For Zevaskyn prior approval, the prescriber must attest that the affected wound has not previously been treated with Zevaskyn.

• Prescriber agreement required: affected wound not previously treated with Zevaskyn.
• Prior approval limits: up to 12 sheets; duration 6 months.

Step-therapy context — supportive care is comparator

Step-therapy context: supportive care (wound care, pain control, infection management, nutrition) is the historical comparator rather than another prior gene therapy.

• Document prior supportive care measures where relevant (wound dressings, infection control, pain management, nutrition).
• There were no prior FDA-approved gene therapies used as a required prior step.

Document prior standard wound care (step from standard care)

Consider documentation of prior standard wound care before advanced therapies; the VIITAL trial compared prademagene to standard-of-care dressings and prior appropriate wound care may be relevant to authorization.

• Document that standard-of-care wound dressings and appropriate local care were tried or considered for the target wound(s).
• For prademagene (VIITAL), control arm received standard wound dressings; use of prior standard care supports alignment with trial populations.

No mandated pharmacologic step therapy in policy excerpts

No formal step therapy algorithm is specified in these excerpts; the policy does not mandate a pharmaceutical prior-step but trials required consideration of standard wound care.

• No explicit prior pharmacologic step therapy requirements present in policy excerpts.
• Documentation of prior standard wound care is suggested but not defined as a required step-therapy drug trial.

Follow product-specific policy criteria prior to approval

Follow the policy’s product-specific criteria and limits for authorization; recent policy additions include operationalized criteria and limits for Zevaskyn and expanded Vyjuvek criteria.

• Refer to the policy sections for product-specific eligibility and limits prior to submitting authorization requests.
• Policy updates (April 2025 additions) added Zevaskyn criteria and Vyjuvek clarifications.

Submit required diagnostic documentation (genetics, NC1, wounds)

Required diagnostic documentation for prior authorization includes genetic confirmation of biallelic COL7A1 mutations, positive NC1 expression in skin, and presence of chronic wound(s) consistent with the listed trial examples.

• Genetic test report documenting biallelic pathogenic COL7A1 mutations.
• Skin biopsy or report showing NC1+ expression where required (per product criteria).
• Wound documentation (size, duration, stage) consistent with chronic wound definitions.

Provide Vyjuvek safety documentation (pregnancy, infection, SCC)

Safety documentation required for Vyjuvek: confirm the patient is not pregnant or breastfeeding and that there is no active infection or active/history of squamous cell carcinoma in the targeted wound(s).

• Document pregnancy and breastfeeding status.
• Document absence of active infection in targeted wound(s).
• Document absence of active or prior squamous cell carcinoma at targeted wound(s).

Document intended therapy details and expected treated area

Include therapy-specific procedural details in clinical documentation: for Vyjuvek, planned topical application and dosing; for Zevaskyn, autologous cell manufacturing and planned surgical application and expected treated area/cycles.

• Describe intended administration: topical Vyjuvek dosing per label; Zevaskyn surgical application and number of sheets to be used.
• For Zevaskyn, document expected treated area and planned number of cycles if relevant to approval limits.

Provide comprehensive clinical documentation (age, wounds, prior care)

Required clinical documentation should include patient age, genetic confirmation or RDEB diagnosis, wound characteristics (size, location, duration, stage), and prior treatments to match pivotal trial populations.

• Age consistent with studied populations (e.g., Vyjuvek ≥6 months; VIITAL enrollment criteria vary).
• Wound characteristics: area, duration, stage, and location.
• Prior treatments and supportive care measures documented in the medical record.

Document wound size, duration, and stage explicitly

Document wound size (area ≥20 cm2), duration (present for ≥6 months), and stage (Stage 2 open skin wound with partial-thickness loss of dermis) when applicable to the requested treatment.

• Wound area: ≥20 cm2 is repeatedly specified in trial eligibility statements.
• Wound duration: present for ≥6 months.
• Stage 2 definition: partial-thickness loss of dermis not extending into subcutaneous tissue.

Documentation note: Stage 2 wound definition required

Note that trial descriptions consistently used Stage 2 wounds: define and document Stage 2 wounds as open skin wounds with partial-thickness loss of dermis that have not extended through the dermis into subcutaneous tissue.

• Ensure medical record describes wound depth consistent with Stage 2 definition used in trials.
• Record photographic or descriptive evidence supporting Stage 2 status when available.

Confirm and document complete wound healing on consecutive visits

When using wound healing as an endpoint for reauthorization, confirm complete wound healing at two consecutive visits (at least 2 weeks apart) as specified in trial protocols.

• Complete wound healing defined as re-epithelialization with no drainage or erosion and only minor crusting.
• Confirmation required at two consecutive visits (e.g., 2 weeks apart) per trial endpoints.

Include required coding documentation with PA

Submit appropriate diagnosis and procedure codes with the prior authorization request to support coverage (e.g., Q81.2 for dystrophic epidermolysis bullosa) and the specific J-code for the product administered.

• Include ICD-10-CM diagnosis Q81.2 (Epidermolysis bullosa dystrophica).
• Include relevant HCPCS/J-codes such as J3401 (Vyjuvek) or J3389 (Zevaskyn) where applicable.

Coverage risk: investigational if criteria not met

Beremagene (Vyjuvek) is considered investigational when continuation criteria are not met and both therapies are investigational for indications not explicitly listed; lack of required documentation may lead to investigational/coverage denial.

• If reauthorization criteria (including documented clinical response) are not met, Vyjuvek is considered investigational.
• Use outside the specified RDEB wound indications is investigational per the policy.

Verify benefit exceptions for the member’s plan

Benefit exceptions apply — this policy may not apply to certain lines of business (e.g., Federal Employee Program, Medicare Supplement, Medicare Advantage, Medicaid, and certain self-insured groups), which can affect coverage adjudication.

• Verify member’s line of business and any state/federal mandates before relying on policy coverage determinations.
• Benefit exceptions may result in different review pathways or denials.

Coverage risk if wound or diagnosis outside trial criteria

Treatment requests for wounds that do not meet the trial criteria (e.g., wounds smaller than 20 cm2, present <6 months, or not Stage 2) or lacking genetic confirmation risk denial for failure to meet coverage criteria.

• Use outside studied inclusion criteria (size, duration, genetic confirmation) may be denied.
• Provide robust documentation if requesting coverage outside trial-like populations.

Exclusion: squamous cell carcinoma at treatment site

Presence of current or prior squamous cell carcinoma at the treatment site is an explicit exclusion in trial descriptions and is a basis for denial of coverage.

• Document absence of SCC at targeted site; an active or prior SCC at the site may trigger denial.
• Trials repeatedly listed SCC at the treatment site as an exclusion.

Apply trial exclusion for SCC at treatment site

The pivotal trials explicitly excluded participants with current or history of squamous cell carcinoma at the treatment site; apply this exclusion when evaluating eligibility for treatment.

• Use trial exclusions to inform eligibility determinations for prior authorization.
• If SCC history is present at the site, deny or consider noncoverage per policy exclusion.

Contraindication/exclusion: SCC at targeted wound site

Current or history of squamous cell carcinoma at the treatment site is documented across trial descriptions as an exclusion and would be a contraindication for these therapies; absence of SCC must be documented.

• Ensure medical record explicitly states no active or prior SCC at the wound site.
• If SCC is present or history is documented at the site, the request is expected to be denied.

Coding inclusion disclaimer — codes don’t guarantee coverage

Inclusion of codes in the policy’s coding table is for reference only and does not guarantee coverage or provider reimbursement; coverage decisions remain subject to member-specific benefit plan terms.

• The presence of HCPCS/CPT/ICD codes in the policy does not imply automatic coverage.
• Always verify member benefits and obtain prior authorization per plan requirements.