Modifiedsierra health and lifePolicy 2026D0120J

Gene Therapies for Hemophilia B

Defines medical necessity, eligibility, and administrative requirements for one-time gene therapies Beqvez (fidanacogene elaparvovec-dzkt) and Hemgenix (etranacogene dezaparvovec-drlb) for treatment of congenital Hemophilia B in adults, and describes conditions under which they are considered unproven or not medically necessary. Affects providers requesting authorization and Hemophilia Treatment Centers managing administration and follow-up.

Policy Summary

Payersierra health and life

PolicyGene Therapies for Hemophilia B

Policy CodePolicy 2026D0120J

Change TypeMaterial revisions to coverage criteria and prior authorization operational checks

Effective DateJan 1, 2026

Next Review Date

Key ActionObtain prior authorization and document eligibility including diagnosis, prophylaxis history, antibody status, and confirm no planned inpatient admission solely for administration.

SourceLink

POLICY UPDATE CHANGES

Added criterion requiring the provider does not request a planned inpatient admission for the sole purpose of administering Beqvez/Hemgenix.

Replaced requirement referencing chronic prophylactic Hympavzi therapy with a broader requirement that the patient is currently receiving routine prophylaxis for hemophilia B with a non-factor replacement therapy (examples listed).

Updated References section to reflect most current information.

one-timeauthorized treatment per lifetime

45 daysauthorization duration from approval

>=18

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Coverage Criteria for Beqvez and Hemgenix

Beqvez (fidanacogene elaparvovec-dzkt) Initial Therapy

Covered when ALL of the following are met:

Beqvez eligibility: Patient is 18 years of age or older; AND one of the following severity presentations: (a) diagnosis of severe hemophilia B with endogenous factor IX < 1% (< 0.01 IU/mL), OR (b) diagnosis of moderately severe hemophilia B with endogenous factor IX ≥ 1% to ≤ 2% (≥ 0.01 IU/mL to ≤ 0.02 IU/mL) and either current or historical life‑threatening hemorrhage or repeated, serious spontaneous bleeding episodes; AND one of the following: (a) patient is currently receiving routine prophylaxis for hemophilia B with a non‑factor replacement therapy (examples: Alhemo [concizumab‑mtci], Hympavzi [marstacimab‑hncq], Qfitlia [fitusiran]), OR (b) patient currently uses factor IX prophylaxis therapy and has had a minimum of 50 exposure days to a factor IX agent; OR patient has been determined to be an appropriate candidate for Beqvez by an HTC based on willingness to adhere to initial and long‑term monitoring and management; AND patient does not have a history of inhibitors to factor IX ≥ 0.6 Bethesda units (BU) and does not screen positive for active inhibitors (≥ 0.6 BU) prior to administration; AND patient does not have neutralizing antibodies to AAVRh74var capsid as detected by an FDA‑approved test; AND patient has not undergone Immune Tolerance Induction (ITI); AND liver health assessments (ALT, AST, ALP, total bilirubin and hepatic ultrasound and/or elastography) rule out radiological liver abnormalities and/or sustained liver enzyme elevations; AND patient is not currently using antiviral therapy for hepatitis B or C; AND HIV negative or, if HIV positive, virally suppressed on antiviral therapy (< 200 copies/mL); AND hepatitis B surface antigen (HBsAg) negative; AND HCV antibody negative OR HCV antibody positive with HCV RNA negative; AND patient has not previously received Beqvez or another factor IX gene therapy; AND Beqvez is prescribed and managed by a bleeding disorder specialist at a federally designated Hemophilia Treatment Center (HTC); AND prescriber attests that ALT/AST and factor IX activity will be monitored at least weekly for at least 4 months following administration and regularly thereafter per labeling; AND prescriber attests that counseling regarding risks of alcohol consumption has been provided; AND dosing is in accordance with FDA labeling; AND provider does not request a planned inpatient admission for the sole purpose of administering Beqvez; AND authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval.See individual criteria (age ≥18; inhibitor <0.6 BU; ≥50 exposure days when applicable)

Derived from Beqvez coverage criteria in policy (includes 01/01/2026 revision adding no planned inpatient admission).

Hemgenix (etranacogene dezaparvovec-drlb) Initial Therapy

Covered when ALL of the following are met:

Hemgenix eligibility: Patient is 18 years of age or older; AND one of the following severity presentations: (a) diagnosis of severe hemophilia B with endogenous factor IX < 1% (< 0.01 IU/mL), OR (b) diagnosis of moderately severe hemophilia B with endogenous factor IX ≥ 1% to ≤ 2% (≥ 0.01 IU/mL to ≤ 0.02 IU/mL) and either current or historical life‑threatening hemorrhage or repeated, serious spontaneous bleeding episodes; AND one of the following: (a) patient is currently receiving routine prophylaxis for hemophilia B with a non‑factor replacement therapy (examples: Alhemo [concizumab‑mtci], Hympavzi [marstacimab‑hncq], Qfitlia [fitusiran]), OR (b) patient currently uses factor IX prophylaxis therapy and has had a minimum of 50 exposure days to a factor IX agent; OR patient has been determined to be an appropriate candidate for Hemgenix by an HTC based on willingness to adhere to initial and long‑term monitoring and management; AND patient does not have a history of inhibitors to factor IX ≥ 0.6 Bethesda units (BU) and does not screen positive for active inhibitors (≥ 0.6 BU) prior to administration; AND patient has not undergone Immune Tolerance Induction (ITI); AND liver health assessments (ALT, AST, ALP, total bilirubin and hepatic ultrasound and/or elastography) rule out radiological liver abnormalities and/or sustained liver enzyme elevations; AND documentation that the patient has been evaluated for preexisting neutralizing anti‑AAV5 antibodies (measured via the CSL Behring LDT) and does not have high anti‑AAV5 titers associated with lack of response; AND patient is not currently using antiviral therapy for hepatitis B or C; AND HIV negative or, if HIV positive, virally suppressed on antiviral therapy (< 200 copies/mL); AND hepatitis B surface antigen (HBsAg) negative; AND HCV antibody negative OR HCV antibody positive with HCV RNA negative; AND patient has not previously received Hemgenix or another factor IX gene therapy; AND Hemgenix is prescribed and managed by a bleeding disorder specialist at a federally designated Hemophilia Treatment Center (HTC); AND prescriber attests that ALT/AST and factor IX activity will be monitored weekly for at least 3 months following administration and regularly thereafter per labeling; AND prescriber attests that counseling regarding risks of alcohol consumption has been provided; AND dosing is in accordance with FDA labeling; AND provider does not request a planned inpatient admission for the sole purpose of administering Hemgenix; AND authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval.

Initial coverage criteria

Covered when ALL of the following are met (policy-level summary reflected in revisions):

Eligibility for Beqvez/Hemgenix: Adult patient with moderate to severe hemophilia B (congenital factor IX deficiency) who currently uses routine factor IX prophylaxis therapy or currently receives routine prophylaxis for hemophilia B with a non‑factor replacement therapy (examples: Alhemo, Hympavzi, Qfitlia).

Based on FDA labeling and policy revision

Bleeding history: Documented current or historical life‑threatening hemorrhage or repeated, serious spontaneous bleeding episodes.

Per FDA label statements

Antibody status (Beqvez‑specific): For Beqvez, absence of neutralizing antibodies to AAVRh74var capsid as detected by an FDA‑approved test is required per FDA labeling.

FDA‑labeled requirement

Beqvez and Hemgenix are considered unproven and not medically necessary for specific clinical situations. These include: treatment of Hemophilia A; repeat administration of Hemgenix or Beqvez for Hemophilia B; treatment of Hemophilia B after prior receipt of another factor IX gene therapy product; routine combination treatment with chronically administered prophylactic therapy for Hemophilia B; and treatment of patients younger than 18 years of age.

Patients who have preexisting neutralizing anti-AAV antibodies to the relevant capsid may not derive benefit from gene therapy and are addressed in the FDA labeling and policy. For Beqvez, absence of neutralizing antibodies to the AAVRh74var capsid as detected by an FDA-approved test is required. For Hemgenix, the pivotal experience includes examples where high anti-AAV5 titers were associated with lack of circulating Factor IX expression; one Hemgenix-treated patient with a preexisting anti-AAV5 titer of 1:3212 had no Factor IX expression and required resumption of exogenous factor prophylaxis.

Consistent with the exclusions, Beqvez and Hemgenix are regarded as unproven and not medically necessary in the situations listed above (see exclusions). The policy revision also emphasizes operational checks performed at authorization to ensure requests do not fall into these exclusion categories.

Requests that include a planned inpatient admission solely for the purpose of administering Beqvez or Hemgenix are not permitted under the revised coverage criteria. Prior authorization reviewers must verify that the provider is not requesting an inpatient stay for the sole purpose of administration; such requests may trigger denial or be considered not medically necessary for the admission.

Coding, Age and Thresholds

Applicable Codesmixed

J1411	Injection, etranacogene dezaparvovec-drlb, per therapeutic dose.
J1414	Injection, fidanacogene elaparvovec-dzkt, per therapeutic dose.
D67	Hereditary factor IX deficiency

Placeholder - no additional groupsmixed

No codes listed

Codes referenced (none listed)mixed

N/A	No procedure/CPT/HCPCS/ICD codes explicitly listed in this document portion.

Age — minimum age for eligible patients

Minimum age>= 18 years

Source context (Beqvez)Beqvez eligibility requires patient is 18 years of age or older

Source context (Hemgenix)Hemgenix eligibility requires patient is 18 years of age or older

Factor IX inhibitor — exclusion threshold

Exclusion threshold (inhibitor)History of or active inhibitors to factor IX >= 0.6 Bethesda units (BU) excludes eligibility

Pre-administration screeningPatients must not screen positive for active factor IX inhibitors defined as >= 0.6 BU prior to administration

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior Authorization Required. Prior authorization is required before administration. Authorization will be issued for no more than one treatment per lifetime and for a duration not to exceed 45 days from approval.

One lifetime treatment only
Authorization duration: ≤ 45 days from approval
Prior authorization required for Beqvez/Hemgenix (J1414, J1411)

Denial Risk

Pre-existing Neutralizing Antibodies

Pre-existing neutralizing antibodies to the relevant AAV capsid may preclude coverage. Coverage is impacted if anti-AAV capsid neutralizing antibodies are detected by an FDA-approved test (e.g., neutralizing antibodies to AAVRh74var for Beqvez).

Document absence of neutralizing antibodies to the applicable AAV capsid per FDA labeling prior to approval

Background and Context

Hemophilia B (congenital Factor IX deficiency) is an X-linked bleeding disorder caused by deficiency of clotting Factor IX. Severe or moderately severe patients frequently require routine factor IX prophylaxis to prevent spontaneous and life‑threatening bleeding. Beqvez and Hemgenix are single‑dose intravenous gene therapies that deliver a high‑activity Padua variant Factor IX transgene to hepatocytes using adeno‑associated viral capsids (Beqvez: AAVRh74var; Hemgenix: AAV5) with the intent of increasing circulating Factor IX activity and reducing bleeding risk. FDA labeling specifies use in adults with moderate to severe disease who currently use factor IX prophylaxis and have a history of life‑threatening or repeated serious spontaneous bleeding; additional safety considerations include preexisting anti‑AAV antibodies, hepatotoxicity monitoring, and infusion‑related reactions.

Definitions

Gene therapy — definition of gene therapy and delivery vectors

Gene therapy — definitionTherapies that introduce RNA or DNA into target cells or tissues via a delivery vector to change gene expression (in vivo or ex vivo), regulated by FDA Center for Biologics Evaluation and Research

Mechanism overviewAchieves changes in gene expression by replacing, correcting, inactivating, or inserting genes using a vector or delivery system

Regulatory contextGene and cellular therapy products are regulated by FDA Center for Biologics Evaluation and Research as biologics

Beqvez — brief definition and mechanism (one-time IV AAVRh74var, FIX-R338L Padua transgene)

Product identityBeqvez (fidanacogene elaparvovec-dzkt): one-time IV gene therapy

Mechanism