ModifiedUnitedHealthcarePolicy 2025D0094E

Roctavian (Valoctocogene Roxaparvovec‑Rvox)

Policy governing medical benefit coverage and prior authorization criteria for Roctavian gene therapy for adults with severe Hemophilia A treated under UnitedHealthcare Commercial plans.

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (Valoctocogene Roxaparvovec‑Rvox)

Policy CodePolicy 2025D0094E

Change TypeOperational and template updates (material change to HTC prescribing/management requirement)

Effective DateApr 1, 2025

Next Review DateN/A

Key ActionObtain prior authorization with documentation that Roctavian will be prescribed and managed by a bleeding disorder specialist at a federally designated Hemophilia Treatment Center (CDC HTC directory) and that the patient meets FDA eligibility criteria.

SourceLink

POLICY UPDATE CHANGES

Replaced criterion requiring Roctavian be administered within a Hemophilia Treatment Center (HTC) with requirement that Roctavian be prescribed and managed by a bleeding disorder specialist on staff at a federally designated HTC listed in the CDC's HTC directory.

Updated Benefit Considerations section via template update.

≥18min age

<1% FVIIIFVIII threshold

1 lifetimelifetime limit

AAV5 neg required

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CDC HTC dirHTC registry

Coverage Criteria for Roctavian

Initial therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

Age and diagnosis: Patient is 18 years of age or older AND has a diagnosis of severe hemophilia A (endogenous factor VIII activity < 1% of normal)age >=18; FVIII activity <1% (<1 IU/dL)

Supported by chunk 2

Prophylaxis or exposure requirement: Either: patient is currently receiving chronic prophylactic emicizumab (Hemlibra) or marstacimab (Hympavzi) OR currently uses factor VIII prophylaxis OR has had a minimum of 150 exposure days to a factor VIII agent>=150 exposure days or current prophylaxis

Supported by chunks 2 and 3

Inhibitor and immunity status: No history of factor VIII inhibitors >= 0.6 Bethesda units (BU) AND does not have pre-existing immunity to the AAV5 capsid as detected by the FDA‑approved AAV5 DetectCDx AND does not screen positive for active factor VIII inhibitors (>= 0.6 BU)inhibitor <0.6 BU; negative AAV5 DetectCDx

Supported by chunk 4

Prior treatments and contraindications: Has not previously received Roctavian or another gene therapy for hemophilia A in the patient's lifetime; has not undergone immune tolerance induction (ITI); and is not currently using antiviral therapy for hepatitis B or Cno prior gene therapy; no ITI; no antiviral therapy

Supported by chunk 5

Liver and infectious disease assessments: Liver health assessments (ALT, AST, ALP, total bilirubin) and hepatic imaging/elastography performed to rule out radiologic abnormalities or sustained enzyme elevations; patient is not HIV positive; hepatitis B surface antigen negative OR if HCV antibody positive then HCV RNA negativeHBsAg negative; HCV RNA negative if Ab+; liver testing per policy

Supported by chunk 4

Site, monitoring, and authorization limits: Roctavian is prescribed and managed by a bleeding disorder specialist on staff at a federally designated Hemophilia Treatment Center (listed in the CDC HTC directory); prescriber attests that counseling on alcohol abstinence for at least 1 year has been provided and that ALT and factor VIII activity will be monitored weekly for at least 26 weeks and regularly thereafter per the PI; dosing per FDA labeling; authorization limited to one treatment per lifetime and issuance for no longer than 45 days from approvalHTC federal designation; weekly monitoring x >=26 weeks; one lifetime dose; <=45 days authorization

Supported by chunk 5

Initial coverage criteria — Covered when ALL of the following are met

Covered when ALL of the following are met:

FDA indication: Patient is an adult with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity below the threshold specified in the FDA indication) without pre-existing antibodies to AAV5 as detected by an FDA‑approved test

Based on FDA indication (chunk 18)

Provider and center requirement: 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

Replaces prior requirement that therapy be administered within an HTC (chunk 20)

Roctavian is explicitly excluded from coverage for several uses. It is not proven or medically necessary for the treatment of mild or moderate hemophilia A, for the treatment of hemophilia B, for repeat administration of Roctavian for hemophilia A, for routine combination with chronically administered prophylactic hemophilia therapies, for treatment after prior receipt of another factor VIII gene therapy, for patients younger than 18 years, and for patients with elevated AAV5 antibodies.

Patients who have pre-existing antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test are excluded from the FDA-indicated population for Roctavian and therefore are not eligible for coverage under this indication.

Repeat administration of Roctavian is specifically identified as not proven or medically necessary. The policy also notes that use of Roctavian in patients with elevated AAV5 antibodies is not medically necessary, and repeat dose administration is an exclusion from coverage.

No additional conditions beyond those already stated in the policy portion were added under the 'not medically necessary' section in this update; the revision was a template update to the Benefit Considerations section and did not introduce new not‑medically‑necessary criteria.

Billing and Diagnosis Codes

HCPCS CodeHCPCS

J1412

Injection, valoctocogene roxaparvovec-rvox; per ml, containing nominal 2 X 1013 vector genomes

Diagnosis CodeICD-10

D66	Hereditary factor VIII deficiency

No codes listed in this document portionmixed

No codes listed

inv-10: Factor VIII activity — threshold value (< 1% / < 1 IU/dL)

Factor VIII activity threshold< 1% of normal (reported as < 1 IU/dL)

Clinical classificationThreshold defines severe hemophilia A (FVIII activity <1%)

Supporting documentationDocumented endogenous FVIII level must be <1% per policy criteria

inv-11: Factor VIII activity — reference to threshold in FDA indication (< IU/dL)

FDA-indicated FVIII threshold referenceFDA indication references factor VIII activity < IU/dL (interpreted as < 1 IU/dL for severe disease)

Indication scope

Prior Authorization, Documentation, and Provider Requirements

Prior Authorization

Prior Authorization and Authorization Limits

Prior authorization is required. Authorization will be issued for no more than one lifetime treatment and for no longer than 45 days from approval. Documentation supporting eligibility criteria must be submitted with the request.

Authorization limit: one treatment per lifetime; approval period up to 45 days
Submit documentation of all eligibility criteria at time of request

Prior Authorization

Prior Authorization Requirement — Document FDA Indication

Prior authorization must document that the patient meets the FDA-indicated population and the policy's clinical criteria (e.g., adults with severe hemophilia A with factor VIII activity <1% and without pre-existing AAV5 antibodies).

Document patient is an adult (≥18 years) with severe hemophilia A and endogenous factor VIII <1% (<1 IU/dL)
Document absence of pre-existing antibodies to AAV5 by an FDA-approved test

Background

Hemophilia A is an X-linked bleeding disorder caused by deficiency of factor VIII. Severe hemophilia A is defined by very low endogenous factor VIII activity (commonly referenced as < 1% FVIII activity (< 1 IU/dL)) and is associated with frequent spontaneous bleeding. Roctavian is an AAV5‑based gene therapy that delivers a B‑domain deleted human FVIII transgene intended to increase factor VIII expression and reduce bleeding and factor replacement use in the FDA‑indicated adult population without pre‑existing anti‑AAV5 antibodies.

Definitions and Key Terms

inv-21: FVIII activity < 1% of normal (<1 IU/dL) — definition for severe hemophilia A

DefinitionFVIII activity < 1% of normal (reported as <1 IU/dL)

Clinical significanceClassifies severe hemophilia A associated with frequent spontaneous bleeding

ComparisonModerate: 1–5% activity; Mild: 5–40% activity

inv-22: Roctavian — AAV5 capsid vector containing B-domain deleted hFVIII gene therapy

Product descriptionAAV serotype 5 capsid containing DNA encoding B-domain deleted hFVIII‑SQ (valoctocogene roxaparvovec)

MechanismAAV5-based, replication-incompetent gene therapy delivering B-domain deleted human FVIII to increase FVIII expression

Policy Summary

PayerUnitedHealthcare

PolicyRoctavian (Valoctocogene Roxaparvovec‑Rvox)

Policy CodePolicy 2025D0094E

Change TypeOperational and template updates (material change to HTC prescribing/management requirement)

Effective DateApr 1, 2025

Next Review DateN/A

SourceLink

On This Page

Applies to adults with congenital factor VIII deficiency meeting the FVIII activity threshold in the FDA indication

Test requirement contextAbsence of pre-existing anti-AAV5 antibodies per FDA-approved test required in the FDA indication

Documentation Required

Evidence of Pre-existing Antibodies and Inhibitors

Providers must document absence of pre-existing immunity to the AAV5 capsid using the FDA-approved companion diagnostic (AAV5 DetectCDx) and confirm the patient does not have active factor VIII inhibitors (≥ 0.6 Bethesda units will preclude coverage).

Document AAV5 antibody negative by FDA-approved test (AAV5 DetectCDx)
Document factor VIII inhibitor testing: inhibitor level must be < 0.6 BU (not ≥ 0.6 BU)

Denial Risk

Coverage May Be Denied if Patient Does Not Meet Indication

Coverage may be denied when the patient does not meet the FDA-indicated population or the policy's clinical criteria (for example: age <18 years, mild or moderate hemophilia A, presence of elevated AAV5 antibodies, prior FVIII gene therapy, or inhibitors ≥ 0.6 BU).

Denial triggers include: age <18, mild/moderate disease, elevated AAV5 antibodies, prior FVIII gene therapy, inhibitors ≥ 0.6 BU

Documentation Required

Prescriber and Hemophilia Treatment Center (HTC) Federal Designation

Prescriber must be a bleeding disorder specialist on staff at a Hemophilia Treatment Center (HTC) that holds Federal designation. Documentation of HTC federal designation (e.g., listing in the CDC HTC directory) must be provided.

Provide evidence of HTC federal designation (CDC HTC directory listing)
Prescriber must attest to counseling and monitoring commitments (alcohol abstinence counseling; monitoring schedule)

Documentation Required

Provider Must Document Diagnosis and HTC Assessment

Provider must document the diagnosis of severe hemophilia A per FDA criteria and policy: endogenous factor VIII activity <1% and clinical determination by the HTC that the patient is an appropriate candidate and will adhere to required monitoring.

Document endogenous factor VIII level <1% (<1 IU/dL) and diagnosis of severe hemophilia A
Document HTC assessment that patient is an appropriate candidate and agrees to initial and long-term monitoring

Documentation Required

Prophylaxis / Exposure Prerequisites

Prophylaxis/exposure prerequisites must be documented: patient is either currently receiving chronic prophylactic emicizumab (Hemlibra) or marstacimab (Hympavzi) OR currently uses factor VIII prophylaxis therapy, or has had ≥150 exposure days to a factor VIII agent.

Document current chronic prophylactic Hemlibra (emicizumab) or Hympavzi (marstacimab) therapy, or ongoing factor VIII prophylaxis
OR document ≥150 factor VIII exposure days

Step Therapy

Step Therapy — No Specifics Provided

No step therapy specifics are provided in this policy; there is no requirement for prior authorization to follow a step-therapy sequence beyond the clinical eligibility criteria listed.

Policy does not specify step therapy steps or preferred prior agents beyond eligibility criteria

Manufacturing noteProduced using Sf9 insect cells and recombinant baculovirus technology

inv-23: Severe hemophilia A — congenital factor VIII deficiency meeting FDA threshold

DefinitionCongenital factor VIII deficiency meeting the FDA-specified factor VIII activity threshold (adult with factor VIII activity < IU/dL)

PopulationFDA-indicated population: adults with severe hemophilia A without pre-existing anti-AAV5 antibodies

Policy linkagePolicy requires documented FVIII activity below the specified threshold to meet coverage criteria

inv-24: Anti-AAV5 antibody status — pre-existing AAV5 antibodies must be absent per FDA-approved test

Required statusPre-existing antibodies to AAV5 must be absent as detected by an FDA-approved test prior to therapy

Exclusion consequencePresence of anti-AAV5 antibodies excludes the patient from the FDA indication and from coverage under that indication

DocumentationPrior authorization must include negative AAV5 antibody testing per an FDA-approved assay