Gene Therapy for the Treatment of Hemophilia B

POLICY NUMBER

L.5.01.594

Beqvez (fidanacogene elaparvovec-dzkt)

Hemgenix (etranacogene dezaparvovec-drlb)

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

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is usually inherited and caused by a defect in one of the genes that determine how the body makes blood clotting factor VIII or IX. Rarely, hemophilia can be acquired. This can happen if the body forms antibodies that attack the clotting factors in the bloodstream, preventing the clotting factors from working. People born with hemophilia have little or no clotting factor, which is a protein needed for normal blood clotting. These proteins work with platelets, which are small blood cell fragments that form in the bone marrow, to help the blood clot. When blood vessels are injured, clotting factors help platelets stick together to plug cuts and breaks on the vessels and stop bleeding.

The two main types of hemophilia are A and B. People with hemophilia A, also called classic hemophilia or Factor VIII deficiency, are missing or have low levels of clotting factor VIII. About 8 out of 10 people with hemophilia have type A. People with hemophilia B, also called Christmas disease or Factor IX deficiency, are missing or have low levels of clotting factor IX.

Beqvez is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

Hemgenix is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. 

Related medical policies -

• Hemophilia Therapies

POLICY

Beqvez (fidanacogene elaparvovec-dzkt) and Hemgenix (etranacogene dezaparvovec-drlb) are considered not medically necessary as there are other formulary alternatives covered by the Plan for treatment of Hemophilia B.

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

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:

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

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

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

D. 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.

POLICY HISTORY

05/15/2023: New policy added.

07/01/2023: Policy Exceptions updated regarding State Health Plan (State and School Employees) Participants.

01/15/2025: Policy title changed from "Hemgenix (etranacogene dezaparvovec-drlb)" to "Gene Therapy for the Treatment of Hemophilia." Policy description updated regarding hemophilia and indications for Beqvez. Policy statement updated to add Beqvez (fidanacogene elaparvovec-dzkt) as not medically necessary as there are other formulary alternatives covered by the Plan for treatment of Hemophilia B and its place in therapy has not been addressed in treatment guidelines. Sources updated. Code Reference section updated to add HCPCS code J1414.

12/02/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy title changed from "Gene Therapy for the Treatment of Hemophilia" to " Gene Therapy for the Treatment of Hemophilia B." First policy statement revised to remove statement that "place in therapy has not been addressed in treatment guidelines." Sources updated.

SOURCE(S)

1. Beqvez prescribing information. Pfizer Laboratories Div Pfizer Inc. May 2024. Last accessed October 2024.

2. Hemgenix prescribing information. CSL Behring. January 2025. Last accessed September 2025.

3. Hemophilia B. National Organization for Rare Disorders. Last updated October 15, 2024. https://rarediseases.org/rare-diseases/hemophilia-b/ . Last accessed September 2025.

CODE REFERENCE

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

Not Medically Necessary Codes

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