CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Hemgenix (etranacogene dezaparvovec) gene therapy for Hemophilia B

Defines medical necessity, dosing, administration, prior authorization documentation, and coding for one-time Hemgenix (intravenous) gene therapy for adult members with moderately severe or severe congenital Factor IX deficiency across Medicaid, Commercial, and Medicare-Medicaid Plan populations.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyHemgenix (etranacogene dezaparvovec) gene therapy for Hemophilia B

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateMay 1, 2023

Next Review DateN/A

Key ActionSubmit required medical records (chart notes) via the PA web portal or fax to support prior authorization requests, including diagnosis confirmation, testing, and monitoring documentation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

one doseAuthorization length (non-renewable)

>=18Minimum patient age for coverage

2 x 10^13 gc/kgRecommended single dose

J1411Primary billable HCPCS code

Medical Necessity Criteria

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Initial Approval Criteria — Hemophilia B (Congenital Factor IX Deficiency)

Coverage is provided when ALL of the following are met. Submission of medical records (chart notes) related to the medical necessity criteria is REQUIRED on all requests.

ALL of the following

Patient is at least 18 years of age
Hemgenix is prescribed by, or in consultation with, a hematologist
Patient will undergo treatment at a manufacturer approved Qualified Treatment Center (QTC)
Patient has a diagnosis of moderately severe or severe congenital Factor IX deficiency (≤ 2% of normal circulating FIX) confirmed by blood coagulation testing and is on continuous routine factor IX prophylaxis unless contraindicated or intolerant
Continuous routine prophylaxis is defined as intent of treating with a predefined frequency of infusions documented in medical records.
Patient has NOT received prior hemophilia AAV-vector-based gene therapy (example: fidanacogene elaparvovec)
ONE of the following clinical bleeding criteria
- Currently use Factor IX prophylaxis therapy (examples provided in policy)
- Have current or historical life-threatening hemorrhage
- Have repeated, serious spontaneous bleeding episodes (e.g., intramuscular hematomas requiring hospitalization, hemarthrosis, CNS bleeding including intracranial hemorrhage, pulmonary hemorrhage, life‑threatening GI hemorrhage, umbilical cord bleeding)
Negative Factor IX inhibitor titers (< 0.6 Bethesda Units) and no prior history of inhibitors
If initial test positive, re-test in ~2 weeks; if re-test also positive, Hemgenix should not be given

Additional Safety/Monitoring and Treatment Conditions (required conditions)

Treatment and monitoring requirements that must be met and maintained:

ALL of the following

Factor IX activity will be monitored periodically (example: weekly for 3 months)
Patients will continue to require exogenous Factor IX until response to Hemgenix occurs
Patients will continue to require exogenous Factor IX until response to Hemgenix occurs
Monitor for presence of inhibitors if bleeding is not controlled
Patient will discontinue Factor IX prophylaxis therapy upon achieving FIX levels of 5% from Hemgenix-derived FIX
Patient has been screened for anti-AAV5 antibody titers and deemed suitable (assay used in HOPE-B trial available via CSL Behring)

Exclusions / Renewal

Limits on coverage and renewal:

ALL of the following

Coverage will be provided for one dose and may not be renewed

Billing and Diagnosis Codes

Covered ICD-10 Diagnosis CodesICD-10Covered

D67	Hereditary factor IX deficiency

HCPCS / BillingHCPCSCovered

J1411

Injection, etranacogene dezaparvovec-drlb, per therapeutic dose; 1 billable unit = 1 kit (weight-based)

NDC (Hemgenix kit sizes by patient weight)NDCCovered

00053-0100-10	Patient weight 46-50 kg — Total volume 100 mL (kit 10)
00053-0110-11	Patient weight 51-55 kg — 110 mL (kit 11)
00053-0120-12	Patient weight 56-60 kg — 120 mL (kit 12)
00053-0130-13	Patient weight 61-65 kg — 130 mL (kit 13)
00053-0140-14	Patient weight 66-70 kg — 140 mL (kit 14)
00053-0150-15	Patient weight 71-75 kg — 150 mL (kit 15)
00053-0160-16	Patient weight 76-80 kg — 160 mL (kit 16)
00053-0170-17	Patient weight 81-85 kg — 170 mL (kit 17)
00053-0180-18	Patient weight 86-90 kg — 180 mL (kit 18)
00053-0190-19	Patient weight 91-95 kg — 190 mL (kit 19)

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Authorization, Documentation, and Administration Requirements

Prior Authorization

Prior authorization and medical record submission required

Submission of medical records (chart notes) documenting diagnosis, step therapy, genetic/mutational testing and other clinical markers is REQUIRED for all authorization requests. Records will be reviewed at the time of submission via the PA web portal or fax.

Include HCPCS code J1411 on the request

Documentation Required

Specialist prescribing and treatment center requirement

Hemgenix must be prescribed by, or in consultation with, a hematologist and the infusion must occur at a manufacturer‑approved Qualified Treatment Center (QTC).

Include HCPCS code J1411 on documentation

Documentation Required

Laboratory testing and monitoring

Providers must document pre‑treatment anti‑AAV5 antibody screening (assay used in HOPE‑B available via CSL Behring), baseline liver function tests, periodic FIX activity monitoring (example: weekly for 3 months), inhibitor testing if bleeding persists, and ongoing hepatic surveillance (abdominal ultrasound and regular AFP monitoring) for patients with HCC risk factors.

Include HCPCS code J1411 on laboratory and monitoring documentation

Denial Risk

Ineligibility if prior AAV-based hemophilia gene therapy or persistent inhibitor

Requests will be denied when the patient has received prior hemophilia AAV‑vector gene therapy or has confirmed positive Factor IX inhibitor titers on repeat testing (re‑test in ~2 weeks).

Affected codes: J1411, D67

Billing Rule

Single-dose preparation and administration constraints

Dose calculation, vial rounding, sterile preparation, infusion technique, and administration must follow product instructions: prepare using aseptic technique; single‑dose intravenous infusion only; DO NOT administer as IV push/bolus; DO NOT infuse diluted Hemgenix in the same IV line with other products; DO NOT use a central line or port.

Include HCPCS code J1411 on administration/billing
Follow weight‑based dose calculation and vial rounding per product labeling

Clinical Background

Hemgenix (etranacogene dezaparvovec) is a one-time, weight‑based AAV5 gene therapy indicated for adults with moderately severe or severe Hemophilia B (congenital factor IX [FIX] deficiency). The HOPE‑B phase 3 trial in adults demonstrated clinical benefit, including a reduction in annualized bleeding rates (from a mean ABR of 4.1 during the lead‑in period to 1.9 during months 7–18 after treatment) and reduction or discontinuation of routine prophylactic FIX in the majority of treated patients (94% discontinued prophylaxis and remained free of previous continuous routine prophylaxis therapy).

Common adverse events observed in the HOPE‑B trial and noted in product literature include liver enzyme elevations, headache, and infusion‑related or flu‑like symptoms.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyHemgenix (etranacogene dezaparvovec) gene therapy for Hemophilia B

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateMay 1, 2023

Next Review DateN/A

Key ActionSubmit required medical records (chart notes) via the PA web portal or fax to support prior authorization requests, including diagnosis confirmation, testing, and monitoring documentation.

SourceLink

On This Page

OpenPayer

Hemgenix (etranacogene dezaparvovec) gene therapy for Hemophilia B

Medical Necessity Criteria

Billing and Diagnosis Codes

Authorization, Documentation, and Administration Requirements

Clinical and Dosing Thresholds

Clinical Background

Defined Terms

Policy Dates