CurrentUnitedHealthcarePolicy IEXD0094.06

Roctavian (valoctocogene roxaparvovec) for Hemophilia A — Coverage Criteria

Medical necessity and coverage criteria for Roctavian gene therapy to treat severe Hemophilia A in adults, including eligibility, required testing, monitoring, and administrative limits for UnitedHealthcare members.

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (valoctocogene roxaparvovec) for Hemophilia A — Coverage Criteria

Policy CodePolicy IEXD0094.06

Change TypeNo material change

Effective DateApr 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization; authorization is limited to one treatment per lifetime and valid for no more than 45 days from approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18+minimum patient age (years)

<1%endogenous FVIII level

150minimum exposure days

1authorization limit

J1412listed HCPCS

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage Criteria for Roctavian (valoctocogene roxaparvovec)

Initial Therapy — Covered when ALL of the following are met:

Covered when ALL of the following are met:

Age: Patient is 18 years of age or older

Diagnosis and severity: Diagnosis of severe hemophilia A AND documentation of endogenous factor VIII levels <1% (<0.01 IU/mL, <1 IU/dL)<0.01 IU/mL

Prior therapy / candidacy: One of: (1) Patient is currently receiving chronic prophylactic emicizumab (Hemlibra) or marstacimab (Hympavzi); OR (2) Patient currently uses factor VIII prophylaxis AND has had a minimum of 150 exposure days to a factor VIII agent; OR (3) Patient has been determined to be an appropriate candidate for Roctavian by the Hemophilia Treatment Center based on willingness to adhere to initial and long-term monitoring and management>=150 exposure days when applicable

Inhibitor history and screening: No history of inhibitors to factor VIII ≥0.6 Bethesda units (BU) AND no active inhibitors on screening (≥0.6 BU)<0.6 BU

AAV5 immunity: No pre-existing immunity to the AAV5 capsid as detected by the FDA-approved companion diagnostic AAV5 DetectCDxnegative on AAV5 DetectCDx

Immune tolerance induction and prior gene therapy: Patient has not undergone Immune Tolerance Induction (ITI) AND has not previously received Roctavian or other gene therapy for hemophilia A

Liver health and viral status: Liver enzyme testing (ALT, AST, ALP, total bilirubin) and hepatic ultrasound/elastography performed to rule out radiologic abnormalities and sustained enzyme elevations; hepatitis B surface antigen (HBsAg) negative; and either HCV antibody negative OR HCV antibody positive with HCV RNA negative; patient is not currently using antiviral therapy for hepatitis B or CHBsAg negative; if HCV Ab positive then HCV RNA negative

HIV: either HIV negative OR HIV positive and virally suppressed (<200 copies/mL)

Treatment setting and monitoring attestation: Roctavian prescribed and managed by a bleeding disorder specialist at a federally designated Hemophilia Treatment Center and prescriber attests to weekly ALT and factor VIII activity monitoring for at least 26 weeks and regular monitoring thereafter per the prescribing information; prescriber attests that counseling has been provided to abstain from alcohol for at least one year following administrationweekly monitoring x >=26 weeks

Dosing and authorization limits: Dosing in accordance with FDA-approved labeling; authorization limited to no more than one treatment per lifetime and valid for no longer than 45 days from approvalone lifetime treatment

Exclusionary clinical conditions relevant to Roctavian include active infection and significant liver disease. Per the policy and supporting clinical trial criteria, patients with evidence of significant liver dysfunction or cirrhosis (including radiologic abnormalities or sustained enzyme elevations), an abnormal liver biopsy showing advanced fibrosis (stage 3–4 within prior 3 years), or laboratory abnormalities such as ALT, bilirubin, or alkaline phosphatase elevations greater than three times the upper limit of normal are excluded. Patients with a positive hepatitis B surface antigen (HBsAg) or a detectable hepatitis C viral load (HCV RNA positive) are not eligible; if HCV antibody is positive the HCV RNA must be negative. Active infections or immunosuppressive disorders are also exclusionary, as are thrombocytopenia with platelet count <100,000/dL and renal impairment at or above the thresholds used in trials (serum creatinine ≥1.4–1.5 mg/dL).

Patients with evidence of pre-existing immunity to the AAV5 capsid are not eligible for Roctavian. The policy requires a negative result on the FDA-approved AAV5 DetectCDx companion diagnostic to proceed. Additionally, a history of factor VIII inhibitors ≥ 0.6 BU or a current screen positive for active FVIII inhibitors (≥ 0.6 BU) disqualifies the patient from treatment. These exclusions mirror trial enrollment criteria that required no prior inhibitor development and absence of detectable anti-AAV5 antibodies.

Applicable Codes and Key Clinical Values

Applicable HCPCSHCPCS

J1412

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

Applicable ICD-10 DiagnosisICD-10

D66	Hereditary factor VIII deficiency

Endogenous FVIII activity

Endogenous FVIII activity requirementDocumentation of endogenous factor VIII levels less than 1% of normal (<0.01 IU/mL; <1 IU/dL)

Associated criterionRequired for diagnosis of severe hemophilia A when assessing eligibility for Roctavian

Measurement contextUsed as part of initial eligibility criteria for Roctavian in adults (age ≥18)

Provider Requirements, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for Roctavian. Authorization limits: one lifetime treatment per patient and authorization duration not to exceed 45 days from approval.

Authorization limit: 1 treatment per lifetime
Authorization duration: ≤ 45 days from approval
Prior authorization required before administration

Denial Risk

Denial Risk Conditions

Do not approve therapy for patients with a history of factor VIII inhibitors ≥ 0.6 Bethesda units (BU) or for patients who screen positive for active inhibitors (≥ 0.6 BU). Also deny if the patient has pre-existing immunity to the AAV5 capsid as detected by the FDA‑approved companion diagnostic (AAV5 DetectCDx®) or if the patient previously received Roctavian or another gene therapy for hemophilia A.

History of factor VIII inhibitors ≥ 0.6 BU — denial risk
Active factor VIII inhibitor screen ≥ 0.6 BU prior to administration — denial risk
Pre-existing AAV5 capsid immunity by AAV5 DetectCDx® — denial risk
Prior gene therapy for hemophilia A (any lifetime receipt) — exclusion

Documentation Required

HTC Federal Designation Required

Providers must document that Roctavian will be prescribed and managed by a bleeding disorder specialist on staff at a Hemophilia Treatment Center (HTC) with Federal designation. Evidence of HTC Federal designation must be provided by listing in the CDC HTC directory.

HTC Federal designation evidenced by listing in the CDC's HTC directory
Roctavian to be prescribed and managed by a bleeding disorder specialist on staff at that HTC

Documentation Required

Documentation and Prescriber Attestations

Prescriber attestations and monitoring requirements must be documented prior to authorization. Attestations required: weekly ALT and factor VIII activity monitoring for at least 26 weeks post‑administration and ongoing monitoring per the prescribing information; counseling on alcohol abstinence for at least one year post‑administration; and commitment to assess treatment efficacy (factor VIII expression, breakthrough bleeding, factor VIII utilization, inhibitor development). UnitedHealthcare may request follow‑up assessments (no more frequently than biannually and up to 5 years) for quality purposes.

Prescriber attests to weekly ALT and FVIII activity monitoring for ≥ 26 weeks, then regularly per PI
Prescriber attests counseling patient to abstain from alcohol for ≥ 1 year post‑administration
Prescriber attests to ongoing assessment of efficacy (FVIII expression, breakthrough bleeding, FVIII utilization, inhibitor development)
UnitedHealthcare may request follow‑up documentation biannually, not to exceed 5 years (quality only)

Definitions

Severe Hemophilia A (definition)

DefinitionSevere hemophilia A: factor VIII (FVIII) activity less than 1% of wild type (<1 IU/dL)

Clinical implicationsAssociated with frequent spontaneous bleeding episodes, predominantly in joints and soft tissues, and higher risk of hemorrhagic complications

Contrast with other severitiesModerate: 1–5% FVIII activity; Mild: 5–40% FVIII activity

Roctavian — product description

Product name and genericRoctavian (valoctocogene roxaparvovec-rvox)

Vector and transgeneAAV serotype 5 (AAV5) capsid containing DNA encoding B-domain deleted SQ form of human coagulation factor VIII (hFVIII‑SQ)

ManufactureDerived from naturally occurring adeno-associated virus and produced using Sf9 insect cells with recombinant baculovirus technology

Replication statusReplication-incompetent AAV vector

Background

Hemophilia A is an X-linked bleeding disorder caused by deficiency of coagulation factor VIII. Patients with severe hemophilia A are defined as having endogenous factor VIII activity less than 1% (<0.01 IU/mL; <1 IU/dL), a level associated with frequent spontaneous bleeding and substantial bleeding-related morbidity. Roctavian (valoctocogene roxaparvovec) is an AAV5-based, single-dose gene therapy that delivers a B-domain deleted human FVIII transgene to increase endogenous FVIII expression; clinical trials enrolled adults with severe disease, no prior FVIII inhibitor history, and no detectable AAV5 immunity.