ModifiedCentenePolicy CP.PHAR.466

Valoctocogene Roxaparvovec-rvox (Roctavian) gene therapy for severe congenital hemophilia A

Medical necessity and coverage criteria for a single-dose AAV5-based gene therapy (Roctavian) for adults with severe congenital hemophilia A; applies to Centene-affiliated health plans across listed lines of business.

Policy Summary

PayerCentene

PolicyValoctocogene Roxaparvovec-rvox (Roctavian) gene therapy for severe congenital hemophilia A

Policy CodePolicy CP.PHAR.466

Change TypeMaterial updates to eligibility, liver assessment and PDAC review added

Effective DateJun 29, 2023

Next Review DateN/A

Key ActionSubmit prior authorization with complete clinical documentation and request PDAC review; include baseline liver tests and attestations as required.

SourceLink

POLICY UPDATE CHANGES

Removed 'life-threatening' from 'life-threatening or serious bleed' criterion to avoid misinterpretation.

Added criteria for baseline liver assessments and hepatologist attestation of Roctavian eligibility for abnormal results; added provider attestation of ability to receive corticosteroids/immunosuppression.

Revised FVIII inhibitor level assay threshold to be < 0.6 BU per clinical study protocols.

Added HCPCS code J1412 for valoctocogene roxaparvovec-rvox.

Added exclusion for prior gene therapy per competitor analysis and pivotal trial exclusion criteria.

Added clarification that all requests reviewed under this policy require medical director review and redirected prior authorization reviews to Precision Drug Action Committee (PDAC) Utilization Management Review.

Added qualifier that the 150 EDs criterion applies to members who have had previous FVIII use and removed requirement for documentation of body weight.

single dosemaximum approved administrations per member

6 x 10^13maximum dose per kg (vg)

>=150 EDsminimum FVIII exposure days

<1 IU/dLsevere hemophilia A definition

2 x 10^13nominal vial concentration

J1412added HCPCS code

Coverage Criteria for Roctavian (Valoctocogene roxaparvovec)

Coverage prerequisites (excerpt)

Covered when ALL of the following are met (items present in this part of document):

Medical review pathway: All requests require medical director review and are redirected to the Precision Drug Action Committee (PDAC) Utilization Management Review.

Prior gene therapy exclusion: Member must not have received prior gene therapy (exclusion).

Liver assessment and hepatologist attestation: Baseline liver assessments required; if abnormal, hepatologist attestation of eligibility per prescribing information required.

Immunosuppression capability: Provider must attest that member can receive corticosteroids and/or other immunosuppressive therapy per prescribing information.

FVIII inhibitor testing: FVIII inhibitor level threshold: < 0.6 Bethesda units (BU) on 2 occasions at least one week apart within the last 12 months.< 0.6 BU

Prior FVIII exposure qualifier: 150 exposure days (EDs) criterion applies to members who have had previous FVIII use; documentation of cumulative EDs recommended.150 EDs

Non‑FDA approved indications for valoctocogene roxaparvovec (Roctavian) are not authorized under this policy unless there is sufficient documentation of efficacy and safety per applicable off‑label use policies or an evidence of coverage document. Providers should reference CP.CPA.09 (commercial), HIM.PA.154 (Marketplace), or CP.PMN.53 (Medicaid) when submitting supporting documentation for off‑label requests.

Contraindications to Roctavian include active infections (acute or uncontrolled chronic), known significant hepatic fibrosis (stage 3 or 4) or cirrhosis, and known hypersensitivity to mannitol. These contraindications mirror the product appendices and should be confirmed by recent clinical and laboratory evaluation prior to authorization.

Prior receipt of any gene therapy is an explicit exclusion to coverage under this policy. Requests for members with documented prior gene therapy may be denied consistent with the competitor analysis and pivotal trial exclusion criteria.

Continued or repeat dosing of Roctavian will not be authorized. Roctavian is indicated to be administered as a single dose, and approval is limited to the initial administration only.

Coding information provided in this policy is for informational purposes and does not, by itself, guarantee coverage. Inclusion or exclusion of codes in the policy does not ensure payment. For example, HCPCS code J1412 (injection, valoctocogene roxaparvovec‑rvox, per mL) is noted for provider reference but its presence is informational and not a guarantee of coverage.

Coding and Dosing Information

Policy Identifiermixed

CP.PHAR.466

Policy reference number

Covered HCPCS Codes (informational)HCPCSCovered

J1412

Injection, valoctocogene roxaparvovec-rvox, per ml, containing nominal 2 x 10 13 vector genomes

inv-10: Maximum dose — single IV infusion of 6 x 10^13 vector genomes (vg) per kg.

Maximum dose (per kg)Single IV infusion not to exceed 6 x 10^13 vector genomes (vg) per kg

Administration frequencySingle dose only; subsequent doses will not be covered

Approval durationApproval duration documented as 3 months (one dose only)

inv-11: Minimum exposure days (EDs) — ≥ 150 EDs of prior FVIII concentrate or cryoprecipitate.

Minimum exposure days (EDs)>= 150 exposure days (EDs) of prior FVIII concentrate or cryoprecipitate for members with previous FVIII use

Rationale

Provider Actions, Documentation, and Review Process

Prior Authorization

PDAC review and documentation requirement

Prior authorization is required. All requests reviewed under this policy require Precision Drug Action Committee (PDAC) Utilization Management Review and must include documentation supporting that the member meets all approval criteria. Requests must be escalated to the medical director/PDAC as required; requests not escalated appropriately may be denied or placed on administrative hold.

Refer to CC.PHAR.21 for PDAC process details.
Provider must submit office chart notes, laboratory results, and any other clinical information showing all approval criteria are met.

Documentation Required

Required baseline liver and immunosuppression attestation

Provider must attest that baseline liver assessments have been completed and submit results. If baseline liver tests are abnormal, a hepatologist attestation of Roctavian eligibility is required. Provider must also attest that the member is able to receive corticosteroids and/or other immunosuppressive therapy if clinically indicated.

Background

Roctavian (valoctocogene roxaparvovec‑rvox) is an adeno‑associated virus (AAV) vector–based gene therapy indicated for adults with severe congenital hemophilia A defined as pre‑treatment factor VIII activity < 1 IU/dL, without pre‑existing anti‑AAV5 antibodies. The product is administered as a recommended single IV infusion dosed at 6 x 10^13 vector genomes per kg, with required baseline and post‑administration monitoring including FVIII levels and liver tests; immunosuppression may be required per the prescribing information.

Definitions

inv-26: Exposure day (ED) — A day on which a person with hemophilia has been infused with factor concentrate to treat or prevent a bleed; ED count includes only days...

DefinitionAn Exposure Day (ED) is a day on which a person with hemophilia receives an infusion of factor concentrate to treat or prevent a bleed

Counting ruleThe ED count includes only days on which factor concentrate was actually infused

Use in policyEDs are used to document prior FVIII exposure (e.g., the 150 EDs criterion)

inv-27: Serious bleeding episodes / Spontaneous bleed — Serious bleeding episodes include intracranial, neck/throat, gastrointestinal, joints (hemarthrosis), muscles (deep compartments), or mucosal bleeds.

Examples of serious bleeding sitesIntracranial; neck/throat; gastrointestinal; joints (hemarthrosis); muscles (deep compartments such as iliopsoas, calf, forearm); mucous membranes

Revision History

2025-11-24policy updateLatest

1Q 2026 annual review added a step therapy bypass for Illinois High-Need Members (IL HIM) per IL HB 5395; clarified that the 150 EDs criterion applies only to members with prior FVIII use; removed requirement for documentation of body weight; references reviewed and updated.

2025-11-04process change

Updated Policy/Criteria language to redirect prior authorization reviews to the Precision Drug Action Committee (PDAC) Utilization Management Review.

2025-03-11process change

Policy Summary

PayerCentene

PolicyValoctocogene Roxaparvovec-rvox (Roctavian) gene therapy for severe congenital hemophilia A

Policy CodePolicy CP.PHAR.466

Change TypeMaterial updates to eligibility, liver assessment and PDAC review added

Effective DateJun 29, 2023

Next Review DateN/A

Key ActionSubmit prior authorization with complete clinical documentation and request PDAC review; include baseline liver tests and attestations as required.

SourceLink

On This Page

150 EDs associated with increased immunologic stability and decreased inhibitor risk

ApplicabilityCriterion applies only to members who have had previous FVIII use

inv-12: Severe hemophilia A definition — pre-treatment FVIII level < 1% or activity < 1 IU/dL.

Severe hemophilia A definitionPre-treatment FVIII level < 1% (or FVIII activity < 1 IU/dL)

Age requirementIndication applies to adults (age ≥ 18 years)

AAV5 antibody requirementNo pre-existing anti-AAV5 antibodies detected by an FDA-approved test (per FDA indication)

inv-13: Cumulative FVIII exposure (EDs) — 150 EDs.

Cumulative FVIII exposure (EDs)150 EDs

Clinical significancePatients rarely develop inhibitors after 150 EDs; associated with immunologic stability

DocumentationProviders should document cumulative EDs (endogenous/exogenous) when applicable

inv-14: Dose per body weight — 6 x 10^13 vg/kg as single IV infusion.

Dose per body weight6 x 10^13 vg/kg as a single IV infusion

Maximum dose statementMaximum dose equals recommended dose: 6 x 10^13 vg/kg body weight

Administration notesDose is delivered as a single intravenous infusion; subsequent doses not covered

Baseline liver assessments per product labeling.
Hepatologist attestation required for abnormal baseline liver results.
Provider attestation of ability to receive corticosteroids/immunosuppression.

Documentation Required

Evidence of prior FVIII exposure (150 EDs qualifier)

For members with prior FVIII use, provider must document cumulative FVIII exposure to confirm at least 150 exposure days (EDs). The 150 EDs qualifier increases likelihood of immunologic stability and is applicable only to members who have had previous FVIII exposure.

Documentation of cumulative FVIII exposure (endogenous and/or exogenous) to confirm ≥150 EDs when applicable.
150 EDs qualifier applies only to members with prior FVIII use.

Step Therapy

Step therapy / prior FVIII prophylaxis

Member must have been adherent with routine FVIII prophylaxis for at least 12 months as assessed and documented by the provider and have had at least one serious spontaneous bleed while on routine prophylaxis to meet step therapy requirements for initial treatment.

Adherence with FVIII product use for routine prophylaxis ≥12 months (documented).
At least one serious spontaneous bleeding event while on routine prophylaxis (see Appendix D for definition and sites).
Only applies to initial treatment dose; subsequent doses not covered.

Step Therapy

Step therapy bypass for IL HIM

Per Illinois HB 5395, a step therapy bypass applies for Illinois High-Need Members (IL HIM). For IL HIM requests submitted on or after 1/1/2026, the step therapy requirements above do not apply.

Step therapy bypass for IL HIM effective 01/01/2026 per IL HB 5395.
Document IL HIM status when requesting bypass.

ContextReferenced as 'serious bleeding episodes' for eligibility criteria (see Appendix D)

ImplicationOccurrence of at least one serious spontaneous bleed while on routine prophylaxis is required for certain criteria

inv-28: Serious bleeding episode — Serious bleeding episodes: bleeds in intracranial; neck/throat; gastrointestinal; joints (hemarthrosis); muscles (deep compartments) etc.

DefinitionSerious bleeding episode: bleeds in intracranial; neck/throat; gastrointestinal; joints (hemarthrosis); muscles (deep compartments such as iliopsoas, calf, forearm); or mucous membranes

ReferenceDefined in Appendix D of the policy for use in clinical criteria

inv-29: Spontaneous bleed — Spontaneous bleed: a bleeding episode occurring without apparent cause and not resulting from trauma.

Spontaneous bleed definitionA bleeding episode that occurs without apparent cause and is not the result of trauma

Policy useRequirement: occurrence of at least one spontaneous bleed while on routine prophylaxis for certain approvals

inv-30: ED (Exposed Day) — ED (Exposed Day): a day on which a person with hemophilia receives an infusion of factor concentrate to treat or prevent a bleed; only da...

ED (Exposed Day)An ED is a day on which a person with hemophilia receives an infusion of factor concentrate to treat or prevent a bleed; only days with infusion count toward EDs

Counting clarificationED tally consists solely of days when factor was infused (endogenous/exogenous infusions included)

Role in criteriaUsed to verify prior exposure thresholds such as the 150 EDs requirement

Added clarification that all requests reviewed under this policy require medical director review.

2024-11-18annual review

1Q 2025 annual review noted no significant clinical changes; references reviewed and updated.

2023-10-27coding update

Added HCPCS code J1412 for valoctocogene roxaparvovec-rvox (informational).

2023-06-29material update

Added criteria for baseline liver assessments and hepatologist attestation for abnormal results; added provider attestation for ability to receive corticosteroids/immunosuppression; revised FVIII inhibitor assay threshold to <0.6 BU; added FVIII products for prophylaxis appendix and weight-based dose calculation; updated serious bleed sites per WFH guidance.

2022-05-09criteria revision

Removed 'life-threatening' from the 'life-threatening or serious bleed' criterion to avoid potential misinterpretation.