ModifiedUnitedHealthcarePolicy IEXDO094.06

Roctavian (Valoctocogene Roxaparvovec-Rvox)

UnitedHealthcare Individual Exchange medical benefit drug policy governing coverage criteria, applicable codes, monitoring and administrative requirements for Roctavian for treatment of severe hemophilia A in adults for Individual Exchange plans (excludes MA, NV, NY).

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (Valoctocogene Roxaparvovec-Rvox)

Policy CodePolicy IEXDO094.06

Change TypeRevised coverage criteria

Effective DateApr 1, 2025

Next Review Date

Key ActionEnsure Roctavian is prescribed and managed by a bleeding disorder specialist on staff at a federally designated Hemophilia Treatment Center and document required pre-treatment diagnostics and monitoring attestation.

POLICY UPDATE CHANGES

Revised coverage criteria to replace requirement that Roctavian be administered within a federally designated HTC with requirement that Roctavian be prescribed and managed by a bleeding disorder specialist on staff at a federally designated HTC.

1Treatment per lifetime allowed

>=150Minimum prior exposure days required

<1%Severe hemophilia A FVIII threshold

0.6Inhibitor exclusion threshold (BU)

Coverage Summary

Medically Necessary / Initial Therapy Criteria

Medically Necessary / Coverage Rationale

Roctavian is proven and medically necessary for the treatment of Hemophilia A (factor VIII deficiency) when ALL of the following are met:

ALL of the following

Patient is 18 years of age or older
Diagnosis of severe hemophilia A
ONE of the following (baseline factor VIII / concurrent therapy)
- Documentation of endogenous factor VIII levels less than 1% of normal factor VIII (< 0.01 IU/mL; < IU/dL)
- Patient is currently receiving chronic prophylactic Hemlibra (emicizumab-kxwh) therapy or Hympavzi (marstacimab-hncq) therapy
- Patient currently uses factor VIII prophylaxis therapy
Exposure and candidacy
- Patient has had a minimum of 150 exposure days to a factor VIII agent
- 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
Inhibitor and immunity
- Patient does not have a history of inhibitors to factor VIII greater than or equal to 0.6 Bethesda units (BU)
- Patient does not have pre-existing immunity to the AAV5 capsid as detected by the FDA-approved companion diagnostic test AAV5 DetectCDx
- Patient does not screen positive for active factor VIII inhibitors as defined as greater than or equal to 0.6 Bethesda units (BU) to administration of Roctavian
- Patient has not gone through Immune Tolerance Induction (ITI)
Liver assessment requirement (ONE of the following)
- Liver health assessments including enzyme testing [alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP) and total bilirubin)] and hepatic ultrasound and elastography are performed to rule out radiological liver abnormalities and/or sustained liver enzyme elevations
HIV and viral suppression (ONE of the following)
- Patient is not HIV positive
- Patient is HIV positive and is virally suppressed with anti-viral therapy (i.e., < 200 copies of HIV per mL)
Hepatitis screening and therapy status
- The patient's hepatitis B surface antigen is negative
- Patient's hepatitis C virus (HCV) antibody is negative OR Patient's HCV antibody is positive and the patient's HCV RNA is negative
- The patient is not currently using antiviral therapy for hepatitis B or C
Patient has not previously received treatment with Roctavian or other gene therapy product for the treatment of hemophilia A in the patient's lifetime
Prescriber attests that the patient's ALT and factor VIII activity will be monitored weekly for at least 26 weeks following administration of Roctavian and regularly thereafter per the monitoring schedule recommended in the prescribing information
Roctavian is prescribed and managed by a bleeding disorder specialist on staff at a Hemophilia Treatment Center (HTC) that holds Federal designation as evidenced by being listed within the CDC's HTC directory
Prescriber attests that counseling has been provided to the patient to abstain from consuming alcohol for at least one year following administration of Roctavian and regarding acceptable alcohol consumption in the longer term
Authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval
Roctavian dosing is in accordance with the United States Food and Drug Administration approved labeling

Not Medically Necessary / Exclusions

Roctavian is not proven or medically necessary for:

ANY of the following

The treatment of mild or moderate hemophilia A
The treatment of hemophilia B
The repeat administration of Roctavian for the treatment of hemophilia A
The routine combination treatment with chronically administered prophylactic therapy for hemophilia A
The treatment of hemophilia A after previously receiving another factor VIII gene therapy product
The treatment of hemophilia A in patients less than 18 years of age
The treatment of hemophilia A in patients with elevated AAV5 antibodies

BU (Bethesda units): laboratory measure of factor VIII inhibitor titer; policy excludes history or active inhibitor >= 0.6 BU.

Exposure days: prior days of exposure to factor VIII concentrate or cryoprecipitate; the policy requires a minimum of 150 exposure days.

AAV5 DetectCDx: the FDA‑approved ARUP Laboratories total antibody assay used to detect pre‑existing antibodies to the AAV5 capsid; a positive result excludes eligibility for Roctavian.

Provider Actions & Administrative Requirements

Prior Authorization

Prior authorization and lifetime limit

Authorization will be issued for no more than one treatment per lifetime and for no longer than 45 days from approval.

J1412

Documentation Required

Pre-treatment documentation and diagnostics

Required pre-treatment documentation and diagnostics include: age 18 years or older; diagnosis of severe Hemophilia A; documentation of endogenous factor VIII activity <1% (or documentation of qualifying concurrent prophylactic therapy such as emicizumab or marstacimab); a minimum of 150 exposure days to a factor VIII agent; negative result on the FDA‑approved AAV5 DetectCDx test (no pre-existing AAV5 antibodies); inhibitor testing showing less than 0.6 Bethesda units (history and active screen); liver health assessments (ALT, AST, ALP, total bilirubin) and hepatic imaging/elastography to rule out radiologic abnormalities or sustained enzyme elevations; and hepatitis B, hepatitis C, and HIV status per policy (including HIV viral suppression definition if applicable).

Age ≥ 18 years
Severe Hemophilia A diagnosis
Endogenous FVIII < 1% of normal (or qualifying concurrent prophylactic therapy)
≥ 150 exposure days to factor VIII
Negative AAV5 DetectCDx (no pre-existing AAV5 antibodies)
No history/active inhibitor ≥ 0.6 BU (< 0.6 BU required)
Liver enzymes (ALT, AST, ALP, total bilirubin), hepatic ultrasound and elastography
Hepatitis B surface antigen negative; HCV antibody negative or HCV RNA negative; HIV status (if HIV+, virally suppressed <200 copies/mL)

Documentation Required

Prescriber and site requirements

Prescriber and site requirements: Roctavian must be prescribed and managed by a bleeding disorder specialist on staff at a Hemophilia Treatment Center (HTC) that holds Federal designation as evidenced by being listed in the CDC HTC directory. The prescriber must attest that counseling has been provided to the patient to abstain from alcohol for at least one year following administration and regarding acceptable longer-term alcohol use, and attest to the patient's willingness to adhere to monitoring and management.

Documentation Required

Post-treatment monitoring attestation

Prescriber must attest that the patient's ALT and factor VIII activity will be monitored weekly for at least 26 weeks following administration of Roctavian and regularly thereafter according to the monitoring schedule recommended in the prescribing information.

Documentation Required

Quality follow-up data

UnitedHealthcare may request follow-up documentation for quality purposes, including evaluation of factor VIII expression, breakthrough bleeding episodes, factor VIII product utilization, and inhibitor development while the patient is under the prescriber's care. Such requests will not be made more frequently than biannually and will not exceed a period of 5 years.

Denial Risk

Denial risk if exclusion criteria met

Claims may be denied if exclusion criteria are met. Common denial scenarios include: patient younger than 18 years; mild or moderate hemophilia A; prior receipt of Roctavian or other gene therapy; presence of pre-existing or elevated AAV5 antibodies (positive AAV5 DetectCDx); active hepatitis B or C or unsuppressed HIV (or not meeting viral suppression criteria); history of inhibitors or active inhibitor ≥ 0.6 BU; prior Immune Tolerance Induction (ITI).

Clinical Evidence Summary

Roctavian is an AAV5‑based, replication‑incompetent gene therapy that delivers a DNA sequence encoding the B‑domain deleted hFVIII‑SQ (valoctocogene roxaparvovec) to enable endogenous factor VIII expression. Clinical development includes phase 1/2 dose‑escalation studies and a phase 3 open‑label, single‑dose trial. Studies evaluated single intravenous administration in adult males with severe hemophilia A, required absence of pre‑existing AAV5 antibodies (screened with the FDA‑approved AAV5 DetectCDx), and required prior exposure to factor VIII (minimum 150 exposure days in trials). Multi‑year follow‑up showed substantial reductions in annualized bleeding rates (ABR) and marked decreases in exogenous factor VIII replacement use.

Background & Definitions

Roctavian is a single‑dose AAV5 vector gene therapy encoding B‑domain deleted hFVIII‑SQ intended for adults with severe hemophilia A who meet specified eligibility and monitoring requirements. Use is restricted to patients without pre‑existing AAV5 antibodies and with appropriate liver, viral, and inhibitor screening. Prescribers must be bleeding disorder specialists on staff at a federally designated Hemophilia Treatment Center, and patients require intensive post‑treatment monitoring (including weekly ALT and factor VIII activity for at least 26 weeks) and long‑term follow‑up per the prescribing information.

Term	Definition
Exposure days	Prior days of exposure to factor VIII concentrate or cryoprecipitate; policy requires minimum of 150 exposure days.
BU (Bethesda units)	Laboratory measure of factor VIII inhibitor titer; policy excludes history or active inhibitor greater than or equal to 0.6 BU.
AAV5 DetectCDx	FDA‑approved total antibody assay (ARUP Laboratories) to detect pre‑existing antibodies to the AAV5 capsid used to screen eligibility for Roctavian.

OpenPayer

Roctavian (Valoctocogene Roxaparvovec-Rvox)

Coverage Summary

Medically Necessary / Initial Therapy Criteria

Not Medically Necessary / Exclusions

Provider Actions & Administrative Requirements

Applicable Codes

Clinical Evidence Summary

Background & Definitions

Revision History