Treatment of Epidermolysis Bullosa

POLICY NUMBER

L.5.01.605

Filsuvez (birch triterpenes)

Vyjuvek (beremagene geperpavec-svdt)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Dystrophic epidermolysis bullosa (DEB) is an ultra-rare genetic connective tissue disorder caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. The COL7A1 gene codes for type VII collagen (C7), a major component of structures in the skin called anchoring fibrils found in the epidermal basement membrane located between the epidermis (top layer of skin) and dermis (underlying layer). Mutations in the COL7A1 gene disrupt adhesion of the epidermis to the dermis. Patients with DEB completely lack or are deficient in COL7A1, resulting in skin fragility and multiple recurring wounds that are difficult to manage. Over time, repeated blistering and fibrosis can lead to squamous-cell carcinoma, life-threating infections, and limb deformities. DEB may be inherited as a dominant or recessive trait; generally, recessive DEB (RDEB) is more severe than dominant DEB (DDEB). There is, however, considerable phenotypic overlap between types. DEB affects approximately 9,000 people globally, including approximately 3,000 people in the U.S. and approximately 3,000 in Europe.

Junctional epidermolysis bullosa is an autosomal recessive condition that is caused by mutations in the genes that code for collagen 17 or laminin 332. Collagen 17 and laminin 332 are proteins that help anchor the skin together. Without them the skin separates easily, causing blisters. There are many types of junctional epidermolysis, and all of them cause widespread blistering. Some forms of junctional epidermolysis improve as the patient gets older, while a rare form of junctional epidermolysis can be fatal in infancy. Because this disease is autosomal recessive, parents can carry the genetic mutations and be completely healthy.

Vyjuvek (beremagene geperpavec-svdt) is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

Filsuvez (birch triterpenes) topical gel is indicated for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in adult and pediatric patients 6 months of age and older.

POLICY

The use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements.

Initial Criteria

Vyjuvek (beremagene geperpavec-svdt) may be considered medically necessary when ALL of the following criteria are met:

1. The individual is ≥6 months of age;

2. The individual has a documented diagnosis of dystrophic epidermolysis bullosa (DEB);

3. The provider has submitted clinic chart notes and laboratory values confirming a mutation in the collagen type VII alpha 1 chain (COL7A1) gene;

4. The individual has at least ONE of the following clinical features of DEB:

   1. Blistering wounds; OR

   2. Scarring;

5. The individual has at least one recurrent or chronic open wound that meets ALL of the following:

   1. Adequate granulation tissue;

   2. Excellent vascularization;

   3. No evidence of active wound infection;

   4. No evidence of history of squamous cell carcinoma; AND

   5. No recent (within the past 3 months) skin graft;

6. The prescriber is a specialist or has consulted with a dermatologist with expertise in the treatment of DEB;

7. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

8. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 6 months

Renewal Criteria

Vyjuvek (beremagene geperpavec-svdt) may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has been previously approved for therapy with the requested agent through BCBSMS PA process;

2. The individual has documented clinical improvement (i.e. decrease in wound size, increase in granulation tissue, complete wound closure);

3. Wound(s) being treated meet ALL of the following:

   1. Adequate granulation tissue;

   2. Excellent vascularization;

   3. No evidence of active wound infection;

   4. No evidence of history of squamous cell carcinoma; AND

   5. No recent (within the past 3 months) skin graft;

4. The prescriber is a specialist or has consulted with a dermatologist with expertise in the treatment of DEB;

5. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

6. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 6 months

Filsuvez (birch triterpenes) is considered not medically necessary as other formulary alternatives are covered for the treatment of epidermolysis bullosa.

Services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

Hood Packaging: Gene therapy and all gene therapy related services are excluded from coverage effective 01/01/2025.

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

1. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

2. appropriate with regard to standards of good medical practice; and

3. not solely for the convenience of the Member, his or her Provider; and

4. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

POLICY HISTORY

05/15/2024: New policy added.

09/16/2024: Policy title changed from "Vyjuvek (beremagene geperpavec-svdt)" to "Treatment of Epidermolysis Bullosa." Policy description updated regarding junctional epidermolysis bullosa and indications for Filsuvez (birch triterpenes). Added policy statement that Filsuvez (birch triterpenes) is considered not medically necessary as other formulary alternatives are covered for the treatment of epidermolysis bullosa. Sources updated.

01/15/2025: Policy Exceptions updated to add the following for Hood Packaging: Gene therapy and all gene therapy related services are excluded from coverage effective 01/01/2025.

06/03/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy description updated. Policy statement revised to state that the use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements. Initial and renewal criteria updated with minor changes. Sources updated.

SOURCE(S)

1. Dermatology. (n.d.). Epidermolysis Bullosa Clinic Frequently asked questions. https://med.stanford.edu/dermatology/resources/gsdc/eb_clinic/eb-faqs.html

2. Filsuvez prescribing information. Amryt Pharmaceuticals DAC. February 2024. Last accessed April 2025.

3. Guide SV, Gonzalez ME, et al. Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa. N Engl J Med. 2022 Dec 15;387(24):2211-2219. doi: 10.1056/NEJMoa2206663. PMID: 36516090.

4. Vyjuvek prescribing information. Krystal Biotech, Inc. January 2025. Last accessed April 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

CPT copyright American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.