Background

Beqvez (fidanacogene elaparvovec-dzkt) is a one-time gene therapy developed by Pfizer for treating adults with moderate to severe hemophilia B. It uses a bioengineered adeno-associated virus (AAV) vector to deliver a working copy of the factor IX (FIX) gene, aiming to enable the body to produce its own clotting factor.

Policy (Criteria)

Beqvez (fidanacogene elaparvovec-dzkt) is considered MEDICALLY NECESSARY when the following criteria are met:

1. Patient is 18 years of age or older
2. Prescribed by or in consultation with a hematologist
3. Patient has the diagnosis of hemophilia B meeting one of the following:
   1. Currently use Factor IX prophylaxis therapy, OR
   2. 2. Have current or historical life-threatening hemorrhage, OR
   3. 3. Have repeated, serious spontaneous bleeding episodes, AND
   4. 4. Do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test
4. Patient has moderate to severe disease as defined by factor IX levels ≤ 2% of normal or  <2 IU/dl
5. Patient has had >150 previous exposure days to factor IX
6. Patient does not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test
7. Patient must have no history of inhibitors to factor IX, and a screen performed within two weeks prior to administration must be negative as defined as ≤ 0.5 Bethesda units
8. Patient has had a hepatic ultrasound and elastography performed prior to administration.

Continuation of Beqvez (fidanacogene elaparvovec-dzkt) is considered MEDICALLY NECESSARY when the following criteria is met: No reauthorization will be approved. One infusion per lifetime.

References

1. BeqvezTM (fidanacogene elaparvovec-dzkt) [package insert]. New York, NY: Pfizer, Inc.; April 2024.
2. A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy with PF-0638435 in Adult Males with Moderately Severe to Severe Hemophilia B (BENEGENE-2). Last updated 07/26/2024. Available at the website below: https://clinicaltrials.gov/study/NCT0386127
3. BEQVEZ Code and Cost Information. https://buyandbill.com/beqvez-j1414/

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each Policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, technology assessment program (TEC) and other non-affiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines.

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