CurrentUnitedHealthcarePolicy 2025D0094E

Roctavian (valoctocogene roxaparvovec) for Hemophilia A — Coverage Criteria

This policy defines UnitedHealthcare's medical necessity criteria, coverage limitations, and administrative requirements for Roctavian gene therapy in adults with severe Hemophilia A and the conditions under which prior authorization will be granted.

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (valoctocogene roxaparvovec) for Hemophilia A — Coverage Criteria

Policy CodePolicy 2025D0094E

Change TypeNew policy

Effective DateApril 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and document negative AAV5 DetectCDx, FVIII <1%, inhibitor testing, liver assessments, and prescriber attestations.

SourceLink

POLICY UPDATE CHANGES

Policy added with detailed eligibility, monitoring, and site-of-care requirements for Roctavian in adults with severe hemophilia A.

18+minimum patient age for coverage

<1%FVIII threshold for severe disease

150minimum prior exposure days

1 lifetimeauthorization limit

J1412listed procedure code

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (valoctocogene roxaparvovec) for Hemophilia A — Coverage Criteria

Policy CodePolicy 2025D0094E

Change TypeNew policy

Effective DateApril 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization and document negative AAV5 DetectCDx, FVIII <1%, inhibitor testing, liver assessments, and prescriber attestations.

SourceLink

On This Page

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:

Root: All items below are required unless an OR branch is specified

Initial eligibility for Roctavian

Age and diagnosis: Patient is 18 years of age or older AND diagnosis of severe hemophilia A with endogenous FVIII <1% (<0.01 IU/mL, <1 IU/dL)age >=18; FVIII <1%

Current therapy or exposure: One of: currently receiving chronic prophylactic emicizumab (Hemlibra) or marstacimab (Hympavzi); OR currently uses factor VIII prophylaxis and has >=150 exposure days to a factor VIII agent; OR determined an appropriate candidate by a Hemophilia Treatment Center based on willingness to adhere to monitoring and management>=150 exposure days

Alternative eligibility paths

Inhibitor and immunity exclusions: No history of inhibitors to factor VIII >=0.6 Bethesda units (BU) AND no active factor VIII inhibitors (screen <0.6 BU) prior to administration AND negative for pre-existing AAV5 capsid immunity by FDA‑approved AAV5 DetectCDxinhibitor <0.6 BU; AAV5 DetectCDx negative

Liver and infection requirements: No active hepatitis B (HBsAg negative) AND either HCV antibody negative OR HCV antibody positive with HCV RNA negative; patient must not be currently using antiviral therapy for hepatitis B or C; liver enzyme testing (ALT, AST, ALP, total bilirubin) and hepatic ultrasound/elastography performed to exclude radiologic abnormalities or sustained enzyme elevationsHBsAg negative; HCV RNA negative if Ab+

Other exclusions and HIV status: No prior Immune Tolerance Induction (ITI) AND no prior Roctavian or other gene therapy for hemophilia A in the patient's lifetime; AND patient is either not HIV positive OR if HIV positive is virally suppressed on antiretroviral therapy (<200 copies/mL)HIV <200 copies/mL if positive

Site of care, monitoring, and counseling: Roctavian must be prescribed and managed by a bleeding disorder specialist at a federally designated Hemophilia Treatment Center (listed in the CDC HTC directory); prescriber must attest to weekly monitoring of ALT and factor VIII activity for at least 26 weeks and regular long‑term monitoring per prescribing information; prescriber must attest that patient received counseling to abstain from alcohol for at least one year following administrationweekly ALT and FVIII x26 weeks

Dosing and authorization limits: Dosing per FDA‑approved labeling; authorization limited to one treatment per lifetime and for no longer than 45 days from approval1 lifetime treatment; <=45 days authorization

Exclusionary and contextual conditions that preclude Roctavian include evidence of immune response to the AAV5 capsid, active viral hepatitis, significant liver disease, or active inhibitor/immune conditions. Specifically, the patient must not have pre-existing immunity to AAV5 as detected by the FDA‑approved AAV5 DetectCDx test; patients with detectable antibodies to AAV5 in clinical trials were excluded. Active hepatitis B (positive HBsAg) or active hepatitis C (detectable HCV RNA) are exclusionary, and patients must not be receiving antiviral therapy for hepatitis B or C at the time of administration. Liver assessments including ALT, AST, ALP, total bilirubin, hepatic ultrasound, and elastography must be performed to rule out radiologic abnormalities or sustained enzyme elevations; significant liver dysfunction (for example, elevations >3× upper limit of normal, cirrhosis, or stage 3–4 fibrosis on biopsy) was an exclusion in studies.

Patients with a history of or current factor VIII inhibitors at or above the specified threshold are excluded: clinical criteria require no history of inhibitors ≥ 0.6 Bethesda units (BU) and a pre‑administration inhibitor screen demonstrating active inhibitors 0.6 BU. Other exclusions and contextual conditions include prior Immune Tolerance Induction (ITI), prior receipt of Roctavian or any gene therapy for hemophilia A, platelet count <100,000/dL, elevated serum creatinine above trial thresholds, active infection or immunosuppressive disorders (including uncontrolled HIV), and active malignancy as described in the clinical studies and policy.

Not covered / contraindications: use of Roctavian is not medically necessary when the patient has pre‑existing AAV5 antibodies as demonstrated by the FDA‑approved AAV5 DetectCDx companion diagnostic; a positive DetectCDx test is an explicit noncoverage condition. Similarly, active factor VIII inhibitors at or above 0.6 BU constitute a contraindication to treatment, and a pre‑administration inhibitor screen must show inhibitors 0.6 BU for coverage to be considered.

Additional noncoverage situations include active hepatitis B (HBsAg positive) or active hepatitis C (HCV RNA detectable), prior gene therapy for hemophilia A, recent or prior Immune Tolerance Induction, significant liver disease or radiologic/biopsy evidence of advanced fibrosis or cirrhosis, and other active infections or immunosuppressive conditions described in the policy. Authorization will not be issued if these contraindications are present.

Coding and Clinical Thresholds

Applicable HCPCS CodesHCPCS

J1412

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

Applicable Diagnosis CodesICD-10

D66	Hereditary factor VIII deficiency

inv-06: Endogenous factor VIII activity — clinical threshold defining severe disease

ThresholdEndogenous factor VIII activity less than 1% of normal (< 0.01 IU/mL, < 1 IU/dL)

Clinical implicationMeets definition of severe hemophilia A when endogenous FVIII <1%

Source guidancePolicy requires documentation of endogenous FVIII <1% as part of eligibility for Roctavian

inv-07: Minimum prior exposure days to factor VIII — requirement for patients using factor VIII prophylaxis (>= 150 exposure days)

Requirement>= 150 exposure days to a factor VIII agent (for patients using factor VIII prophylaxis)

ContextApplies when patient currently uses factor VIII prophylaxis as one pathway to eligibility for Roctavian

Prior Authorization, Documentation, and Operational Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Authorization is limited to one Roctavian (valoctocogene roxaparvovec-rvox) treatment per lifetime and, if approved, the authorization is effective for up to 45 days from the date of approval.

Authorization required: 1 treatment per lifetime; authorization effective up to 45 days

Documentation Required

Documentation Requirements

Provider must document the patient's age and diagnosis, including: diagnosis of severe Hemophilia A (Factor VIII deficiency) with endogenous factor VIII level <1% of normal (< 0.01 IU/mL, < 1 IU/dL); prior exposure history or current prophylaxis status; and other required clinical and laboratory information per policy (e.g., inhibitor testing, AAV5 DetectCDx results, liver assessment, HIV/HBV/HCV status).

Document age (patient must be 18 years or older)
Document diagnosis of severe Hemophilia A and endogenous FVIII <1% (< 0.01 IU/mL, < 1 IU/dL)

Background

Hemophilia A is an X‑linked bleeding disorder caused by deficiency of coagulation factor VIII; severe hemophilia A is defined as endogenous factor VIII activity less than 1% (< 0.01 IU/mL, < 1 IU/dL). Patients with severe disease experience frequent spontaneous bleeding and often require lifelong replacement or prophylactic therapies.

Roctavian (valoctocogene roxaparvovec) is an AAV5‑based gene therapy that delivers a B‑domain deleted human FVIII transgene with the intent to increase endogenous FVIII expression, reduce bleeding rates, and decrease the need for exogenous factor VIII. Clinical trials enrolled adults with severe hemophilia A who had no history of inhibitor development, no detectable pre‑existing AAV5 antibodies (screened using the FDA‑approved AAV5 DetectCDx total antibody assay), and, for many cohorts, at least 150 prior exposure days to factor VIII products when on prophylaxis.

Because Roctavian is a one‑time systemic gene therapy, careful patient selection and baseline evaluation—particularly for AAV5 immunity, liver health, viral hepatitis status, and factor VIII inhibitors—are required to optimize safety and efficacy and to satisfy coverage criteria.

Key Definitions

inv-13: Severe Hemophilia A — clinical definition (endogenous FVIII activity <1% / < 0.01 IU/mL)

DefinitionSevere Hemophilia A: endogenous factor VIII activity less than 1% of normal (< 0.01 IU/mL, < 1 IU/dL)

Clinical featuresAssociated with frequent spontaneous bleeding episodes, predominantly in joints and soft tissues

ClassificationModerate: 1–5% FVIII activity; Mild: 5–40% FVIII activity

inv-14: AAV5 DetectCDx — FDA-approved companion diagnostic to detect pre-existing antibodies to AAV5 capsid required for patient selection

Test nameAAV5 DetectCDx (FDA-approved companion diagnostic)

PurposeDetects pre-existing antibodies to the AAV5 capsid to determine patient eligibility for Roctavian