CurrentCommunity-CarePolicy N/A

valoctocogene roxaparvovec (gene therapy) for Hemophilia A

Defines medical necessity, lab and clinical requirements, quantity limits, and authorization period for valoctocogene roxaparvovec for adults with hemophilia A; applies to Medical Benefit medication utilization.

Policy Summary

PayerCommunity-Care

Policyvaloctocogene roxaparvovec (gene therapy) for Hemophilia A

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization with required documentation including hematologist involvement, specified labs and test results, exposure history, and evidence of no prior gene therapy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

18Minimum age for therapy

1Authorized fills per lifetime

1 yrAuthorization period (no renewal)

<0.6 BU x2Inhibitor testing requirement

Medical Necessity Criteria

Medical Necessity Criteria for Hemophilia A (valoctocogene roxaparvovec)

Covered when ALL of the following are met:

Prescribed by, or in consultation with, a hematologist

Diagnosis of hemophilia A (Factor VIII deficiency, classic hemophilia)

Sex requirement

Patient's sex is male
Prescriber has provided information that the requested agent is medically appropriate for the patient's sex
medical records required

Policy Summary

PayerCommunity-Care

Policyvaloctocogene roxaparvovec (gene therapy) for Hemophilia A

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization with required documentation including hematologist involvement, specified labs and test results, exposure history, and evidence of no prior gene therapy.

SourceLink

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Exposure days / prophylaxis

ALL of the following
- Patient is on prophylactic therapy with a Factor VIII agent (e.g., Advate, Eloctate, Recombinate)
- Patient has had a minimum of 150 exposure days
  medical records required
Prescriber has determined that the patient requires improved protection than they are receiving from their current therapy (examples: increased bleeding due to severely damaged joints, need for anticoagulation, elderly with fall risk)
medical rationale/records required

Liver function limits (no significant liver dysfunction)

ALT (alanine transaminase) < 3 times the upper limit of normal
lab results within the past 3 months required
Bilirubin < 3 times the upper limit of normal
lab results within the past 3 months required
Alkaline phosphatase (ALP) < 3 times the upper limit of normal
lab results within the past 3 months required
INR < 1.4
lab results within the past 3 months required

Authorization, Documentation, and Denial Risks

Prior Authorization

Authorization required with documentation

Prior authorization is required and must demonstrate that all medical necessity criteria are met, including specialty involvement, required laboratory and clinical data, exposure history, infection and liver/renal testing, inhibitor testing, and documentation that the patient has not received prior gene therapy.

Affected code: J1412
Required elements: hematologist involvement; Factor VIII baseline level and Nijmegen Bethesda assay results; exposure days/prophylaxis history; HIV/HBV/HCV testing; liver function tests and creatinine; anti‑AAV antibody titers; documentation of no prior gene therapy

Documentation Required

Required lab and clinical records

Provide the specified laboratory and clinical records with the request; many tests must be recent as noted in the criteria (within past 3 months or 12 months where specified).

Factor VIII baseline residual level (lab test required; used to establish disease severity)
Modified Nijmegen Bethesda assay: < 0.6 BU on 2 consecutive occasions ≥ 1 week apart within the past 12 months
HIV testing and, if HIV positive, viral load within past 12 months < 1000 copies/mL
Hepatitis B surface antigen and HCV antibody (and HCV RNA if antibody positive) — lab tests within past 3 months
Liver function tests within past 3 months: ALT, bilirubin, ALP and INR to demonstrate ALT, bilirubin, ALP each < 3× ULN and INR < 1.4
Serum creatinine within past 3 months to confirm creatinine < 1.5 mg/dL
Anti‑AAV antibody titers (e.g., AAV‑5) within past 3 months per labeling limits
Bleed history and exposure days (minimum 150 exposure days if on prophylactic Factor VIII therapy) and prescriber rationale/medical records

Denial Risk

Noncompliance with exclusion criteria

Requests will be denied if the patient meets any exclusion criteria listed in the policy.

Active inhibitors to Factor VIII
Previous gene therapy for hemophilia A (including valoctocogene roxaparvovec)
Any active infection
Significant liver dysfunction (ALT, bilirubin, or ALP ≥ 3× ULN, or INR ≥ 1.4)
Creatinine ≥ 1.5 mg/dL
Anti‑AAV antibody titers that exceed labeling administration instructions (e.g., AAV‑5)

Clinical Thresholds and Authorization Metrics

Screening and laboratory thresholds

Factor VIII baseline residual level≤ 1 IU/dL OR >1 and ≤5 IU/dL with severe-bleed history

Modified Nijmegen Bethesda assay< 0.6 Bethesda Units on 2 consecutive occasions ≥ 1 week apart within past 12 months

Minimum exposure days≥ 150 exposure days (if on prophylactic Factor VIII therapy)

HIV viral load< 1000 copies/mL (if HIV positive; within past 12 months)

Creatinine< 1.5 mg/dL (must NOT be ≥ 1.5 mg/dL; within past 3 months)

Liver enzymes and labsALT, Bilirubin, ALP each < 3x ULN; INR < 1.4 (within past 3 months)

Age ≥ 18Minimum age for therapy

1Authorized fills per lifetime (single kit)

1 yearAuthorization period (no renewal)

< 0.6 BU x2Inhibitor testing requirement (Modified Nijmegen on 2 occasions)

Clinical Background

Valoctocogene roxaparvovec is a single-dose gene therapy delivered as a vector infusion intended to provide Factor VIII expression for people with hemophilia A. When authorized, it is furnished as a one-time lifetime dose (one kit) under the medical benefit and requires prior authorization using HCPCS code J1412.

Eligibility for valoctocogene roxaparvovec requires careful pre-treatment screening to confirm appropriateness and safety. Required evaluations include documented diagnosis and specialist involvement (prescribed by or in consultation with a hematologist), assessment of disease severity (baseline Factor VIII activity thresholds), and documentation of exposure history to Factor VIII replacement when applicable.

Important laboratory and clinical screens include testing for inhibitors to Factor VIII (a modified Nijmegen Bethesda assay result of < 0.6 BU on two occasions ≥ 1 week apart within the past 12 months), evaluation of liver function and renal function (ALT, bilirubin, ALP each must be < 3× ULN and INR < 1.4; creatinine must be < 1.5 mg/dL, with many tests required within the past 3 months), screening for active infections and viral serologies (HIV, hepatitis B surface antigen, and HCV antibody/RNA per requirements), and assessment for anti-AAV antibodies (e.g., AAV‑5) to ensure titers do not exceed labeling administration instructions.

Additional exclusions and documentation requirements include absence of active inhibitors or other active infections, no prior gene therapy for hemophilia A, and demonstration that the therapy is medically appropriate for the patient’s sex and clinical circumstances (for example, patients on prophylactic Factor VIII therapy generally must have ≥ 150 exposure days and/or documented need for improved protection).

OpenPayer

valoctocogene roxaparvovec (gene therapy) for Hemophilia A

Medical Necessity Criteria

Applicable Codes

Authorization, Documentation, and Denial Risks

Clinical Thresholds and Authorization Metrics

Clinical Background

Key Definitions