CurrentCignaPolicy IP0580

Roctavian (valoctocogene roxaparvovec) gene therapy for hemophilia A

Coverage policy for one-time intravenous gene therapy Roctavian for adults with severe hemophilia A administered under Cigna benefit plans; describes prior authorization, clinical criteria, dosing, and documentation requirements.

Policy Summary

PayerCigna

PolicyRoctavian (valoctocogene roxaparvovec) gene therapy for hemophilia A

Policy CodePolicy IP0580

Change TypeMaterial update — new policy creation and multiple revisions

Effective DateOct 10, 2024

Next Review DateN/A

Key ActionRequest prior authorization and submit documentation to support baseline Factor VIII level, AAV5 antibody test result, inhibitor testing, liver labs, platelet count, creatinine, and weight; approval is one-time (per lifetime) if all criteria are met.

SourceLink

POLICY UPDATE CHANGES

New policy created for Roctavian with policy statement, criteria, and documentation requirements.

Updated timing phrases from 'within 30 days before the intended receipt' to 'within the past 30 days' for multiple laboratory and assessment requirements.

Several exclusionary requirements were removed including active infection, immunosuppressive disorder, recent live vaccine, history of thrombosis/thrombophilia, active malignancy, and reproductive precaution language.

Condition 'Prior Receipt of Gene Therapy' was added to Conditions Not Covered (not recommended for approval).

one-timeapproved dosing frequency

6 x 10^13 vg/kgapproved dose

>=150min prior FVIII exposure

<1 IU/dLFVIII activity threshold

Coverage Criteria for Roctavian (valoctocogene roxaparvovec)

Initial Therapy (FDA‑approved indication)

Approve one-time (per lifetime) single dose if ALL of the following criteria A–S are met

FDA-Approved Indication Criteria (A–S)

A–F
- A: Patient is male.
- B: Patient is ≥ 18 years of age.
- C: Patient has not received Roctavian in the past (verification in claims history required). If claims history is unavailable, prescribing physician confirms no prior receipt.
- D: Baseline (without Factor VIII replacement therapy) Factor VIII level < 1 IU/dL (documentation required).
- E: No detectable pre-existing antibodies to adeno-associated virus 5 (AAV5) by an FDA-approved test (documentation required).
- F: History of use of Factor VIII therapy for at least 150 exposure days per the prescribing physician.
G
- G.i: Factor VIII inhibitor titer testing performed within the past 30 days (documentation required).
- G.ii: No current inhibitor to Factor VIII (documentation required).
- G.iii: No history of Factor VIII inhibitors (documentation required).
H: Prophylactic Factor VIII will not be given after Roctavian administration once adequate Factor VIII levels have been achieved (episodic Factor VIII use allowed for treatment of bleeds or surgery as determined by the hemophilia specialist).
I: Patient does not have a known hypersensitivity to mannitol.
J: No chronic or active hepatitis B (documentation required).
K: No active hepatitis C (documentation required).
L: Patient is not human immunodeficiency virus (HIV) positive (documentation required).
M: No evidence of significant hepatic fibrosis or cirrhosis.
N
- N.i: Liver function testing within the past 30 days meets ALL of: a) ALT ≤ 1.25 × ULN; b) AST ≤ 1.25 × ULN; c) Total bilirubin ≤ 1.25 × ULN; d) Alkaline phosphatase ≤ 1.25 × ULN; e) GGT ≤ 1.25 × ULN; f) INR < 1.4 (documentation required).
- N.ii: If one or more of the lab values a–f are outside the specified ranges, a hepatologist has evaluated the patient and determined Roctavian use is clinically appropriate (documentation required).
O: Within the past 30 days, platelet count ≥ 100 × 10^9/L (documentation required).
P: Within the past 30 days, creatinine < 1.4 mg/dL (documentation required).
Q: Medication is prescribed by a hemophilia specialist physician.
R: Current patient body weight obtained within the past 30 days (documentation required).
S: If criteria A through R are met, approve one dose of Roctavian to provide a one-time (per lifetime) single dose of 6 × 10^13 vector genomes per kg by intravenous infusion (verification required).

N/A: None

Any use of Roctavian other than the FDA‑approved indication for hemophilia A is considered experimental, investigational, or unproven. The policy explicitly lists Prior Receipt of Gene Therapy as a condition not covered and notes prior gene therapy was a reason for exclusion in the pivotal study.

Use of Roctavian for indications other than those described in this policy is considered not medically necessary / experimental / investigational. This includes patients with a history of prior gene therapy, which is specified in the policy as a condition not recommended for approval.

Coding and Product Identifiers

Considered Medically Necessary when criteria metHCPCSCovered

J1412

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

Product NDCsNDC

NDC 68135-927-48	Roctavian carton containing one single dose vial
NDC 68135-927-01	Roctavian single dose vial (extractable volume >= 8 mL, contains 16 x 10^13 vector genomes)

inv-06: Platelet count

Platelet count threshold>= 100 x 10^9 /L (within the past 30 days; documentation required)

TimingMeasured within the past 30 days

DocumentationLaboratory report or chart note required to confirm value

inv-07: Creatinine

Serum creatinine threshold< 1.4 mg/dL (within the past 30 days; documentation required)

TimingMeasured within the past 30 days

Provider Requirements, Prior Authorization, and Documentation

Prior Authorization

Prior Authorization and One-Time Approval

Prior Authorization is recommended for Roctavian. Approvals are one-time (per lifetime) for a single dose when all FDA‑approved criteria are met. Because of the specialized evaluation and monitoring required, Roctavian must be prescribed by a hemophilia specialist physician. Verification of certain criteria via claims history or Medical Director review is required as noted in the criteria (e.g., prior receipt of Roctavian, weight-based dosing). All approvals and denials will be forwarded to the Medical Director for evaluation.

One-time (per lifetime) single dose approvals only
Prescribed by a hemophilia specialist physician (Criteria Q)
Verification in claims history required for prior receipt of Roctavian (Criteria C)
Medical Director verification required for dosing (Criteria S)

Step Therapy

Background

Roctavian is an AAV5‑based gene therapy indicated for adults with severe hemophilia A, defined by baseline Factor VIII activity < 1 IU/dL, and requires absence of pre‑existing AAV5 antibodies. Clinical trial data (GENEr8‑1) demonstrated increases in Factor VIII activity and reductions in Factor VIII use and bleeding rates.

Definitions and Key Eligibility Terms

inv-14: Severe hemophilia A

DefinitionSevere hemophilia A: baseline (without Factor VIII replacement) Factor VIII activity < 1 IU/dL

Typical clinical implicationsPatients usually require routine prophylaxis with Factor VIII replacement or emicizumab

PopulationPrimarily affects males (X-linked disorder)

inv-15: AAV5 antibody negative

Required statusNo detectable pre-existing antibodies to adeno-associated virus serotype 5 (AAV5) as determined by an FDA‑approved test

DocumentationResult from an FDA-approved AAV5 antibody test is required

Policy Summary

PayerCigna

PolicyRoctavian (valoctocogene roxaparvovec) gene therapy for hemophilia A

Policy CodePolicy IP0580

Change TypeMaterial update — new policy creation and multiple revisions

Effective DateOct 10, 2024

Next Review DateN/A

SourceLink

On This Page

Laboratory report or chart note required to confirm value

inv-08: Liver labs

ALT/AST/Total bilirubin/ALP/GGTEach ≤ 1.25 x upper limit of normal (within the past 30 days; documentation required)

INR< 1.4 (within the past 30 days; documentation required)

Alternate pathwayIf one or more values exceed these ranges, a hepatologist evaluation documenting clinical appropriateness may satisfy the criterion

Step Therapy / Sequencing

Step therapy / sequencing is not applicable. There is no required prior trial of alternative therapies to obtain Roctavian, however patients must have prior exposure to Factor VIII therapy of at least 150 exposure days as an enrollment/exposure requirement (this is a baseline eligibility requirement, not a step therapy sequence).

Requirement: history of Factor VIII therapy for ≥ 150 exposure days (Criteria F)
This is an eligibility/exposure criterion, not a step-therapy sequencing requirement

Documentation Required

Required Documentation

Documentation is required to support all baseline and safety criteria. Required documentation may include, but is not limited to, chart notes, laboratory results, medical test results, claims records, and prescription receipts. Specific elements that must be documented include prior claims for Roctavian, baseline (off‑therapy) Factor VIII level, AAV5 antibody test results, Factor VIII inhibitor testing and history, liver and hepatitis status, platelet and creatinine values, and current body weight.

Chart notes, labs, test results, claims records, prescription receipts may be requested
Evidence of baseline Factor VIII level < 1 IU/dL (Criteria D)
Documentation of negative AAV5 antibodies by FDA‑approved test (Criteria E)
Factor VIII inhibitor testing within 30 days and no current/history of inhibitor (Criteria G)
Liver function tests and/or hepatologist evaluation as specified (Criteria N)
Platelet count ≥ 100 x10^9/L within 30 days (Criteria O)
Creatinine < 1.4 mg/dL within 30 days (Criteria P)
Current body weight within 30 days (Criteria R)

Denial Risk

Non‑Covered Indications / Prior Gene Therapy

Use of Roctavian for any indication other than the FDA‑approved indication (adult males with severe hemophilia A meeting all criteria) is considered experimental, investigational, or unproven and is not covered. Prior receipt of any gene therapy is specifically a condition not recommended for approval.

Conditions not covered include prior receipt of gene therapy (policy exclusion)
Any non‑FDA‑approved indication for Roctavian is considered experimental/not covered

ConsequencePresence of detectable AAV5 antibodies excludes the patient from meeting this eligibility criterion