Last Review Date: 06/04/2024

Date of Origin: 01/03/2023

Dates Reviewed: 01/2023, 06/2024

1. Length of Authorization

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

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• 1 kit (based on weight chart below)

2. Max Units (per dose and over time) [HCPCS Unit]:

• 1 kit (based on weight chart below)

3. Initial Approval Criteria ^1-13

Coverage is provided in the following conditions:

Hemophilia B (Congenital Factor IX Deficiency) † Ф

• Patient is at least 18 years of age; AND
• Patient has a diagnosis of moderately severe or severe congenital factor IX deficiency bleeding phenotype (i.e., ≤2% of normal circulating factor IX) for which the subject is on continuous routine factor IX prophylaxis, unless there is a contraindication or intolerance (Note: Continuous routine prophylaxis is defined as the intent of treating with an a priori defined frequency of infusions (e.g., twice weekly, once every two weeks, etc.) as documented in the medical records); AND
• Patient has not received prior hemophilia AAV-vector–based gene therapy (e.g., fidanacogene elaparvovec); AND
• Patient has one or more of the following:
  • Currently use Factor IX prophylaxis therapy (e.g., AlphaNine SD, Alprolix, BeneFIX, Idelvion, Ixinity, Mononine, Profilnine, Rebinyn, Rixubis, etc.); OR
  • Have current or historical life-threatening hemorrhage; OR
  • Have repeated, serious spontaneous bleeding episodes (e.g., intramuscular hematomas requiring hospitalization, hemarthrosis, central nervous system (CNS) bleeding (including intracranial hemorrhage), pulmonary hemorrhage, life‐threatening gastrointestinal (GI) hemorrhage and umbilical cord bleeding); AND
• Patient has been tested and found negative for Factor IX inhibitor titers (i.e., <0.6 Bethesda Units) and does not have a prior history of inhibitors (if test result is positive, re-test within approximately 2 weeks. If re-test is also positive, Hemgenix should not be given); AND
• Patient Factor IX activity will be monitored periodically (e.g., weekly for 3 months) as well as presence of inhibitors if bleeding is not controlled (Note: patients will continue to require exogenous Factor IX until response to Hemgenix occurs); AND
• Patient will discontinue Factor IX prophylaxis therapy upon achieving FIX levels of 5% from etranacogene dezaparvovec treatment; AND
• Patient must have a baseline AAV5 Neutralizing antibody (Nab test prior to beginning therapy. (This assay was used in the HOPE-B clinical trial and CSL will provide and cover the expense and shipment of this test); AND
• Patient will have baseline liver function assessed prior to and after therapy according to the monitoring schedule outlined in the product labeling with corticosteroids administered in response to elevations; AND
• Patients with preexisting risk factors for hepatocellular carcinoma (e.g., patients with cirrhosis, advanced hepatic fibrosis, hepatitis C or B, non-alcoholic fatty liver disease (NAFLD), chronic alcohol consumption, non-alcoholic steatohepatitis (NASH), and advanced age) will have abdominal ultrasound screenings and be monitored regularly (e.g., annually) for alpha-fetoprotein (AFP) elevations following administration

† FDA Approved Indication(s); ‡ Compendium Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ¹

Coverage cannot be renewed.

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS code:

• J1411 – Injection, etranacogene dezaparvovec-drlb, per therapeutic dose; 1 billable unit = 1 kit (based on weight chart below)

NDC(s):

Hemgenix kit sizes:

7. References

1. Hemgenix [package insert]. King of Prussia, PA; CSL Behring, LLC., December 2022. Accessed May 2023.
2. MASAC Recommendations Concerning Products Licensed for the Treatment of Hemophilia and Selected Disorders of the Coagulation System. National Hemophilia Foundation. MASAC Document #284; April 2024. Available at: https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-284-masac-recommendations-concerning-products-licensed-for-the-treatment-of-hemophilia-and-selected-disorders-of-the-coagulation-system. Accessed May 2024.
3. Guidelines for the Management of Hemophilia. 3^rd Edition. World Federation of Hemophilia 2020. Available at: https://www1.wfh.org/publications/files/pdf-1863.pdf. Accessed May 2024.
4. Graham A1, Jaworski K. Pharmacokinetic analysis of anti-hemophilic factor in the obese patient. Haemophilia. 2014 Mar;20(2):226-9.
5. Croteau SE1, Neufeld EJ. Transition considerations for extended half-life factor products. Haemophilia. 2015 May;21(3):285-8.
6. Mingot-Castellano, et al. Application of Pharmacokinetics Programs in Optimization of Haemostatic Treatment in Severe Hemophilia a Patients: Changes in Consumption, Clinical Outcomes and Quality of Life. Blood. 2014 December; 124 (21).
7. MASAC RECOMMENDATION CONCERNING PROPHYLAXIS FOR HEMOPHILIA A AND B WITH AND WITHOUT INHIBITORS. National Hemophilia Foundation. MASAC Document #267 (Replaces Document #241); March 2022. Available at: https://www.bleeding.org/sites/default/files/document/files/267_Prophylaxis.pdf. Accessed May 2024.
8. Rayment R, Chalmers E, Forsyth K, et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B. B J Haem:190;5, Sep 2020. https://doi.org/10.1111/bjh.16704.
9. Peyvandi F, Palla R, Menegatti M, et al. Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders. J Thromb Haemost. 2012;10:615‐621.
10. Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644.
11. Pipe S, van der Valk P, Verhamme P, et al. Long-term bleeding protection, sustained FIX activity, reduction of FIX consumption and safety of hemophilia B gene therapy: results from the HOPE-B trial 3 years after administration of a single dose of etranacogene dezaparvovec in adult patients with severe or moderately severe hemophilia B. Blood (2023) 142 (Supplement 1): 1055. https://doi.org/10.1182/blood-2023-187624
12. MASAC Recommendations on Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia. National Hemophilia Foundation. MASAC Document #282. October 2023. Available at: https://www.bleeding.org/healthcare-professionals/guidelines-on-care/masac-documents/masac-document-282-masac-recommendations-on-hemophilia-treatment-center-preparedness-for-delivering-gene-therapy-for-hemophilia. Accessed May 2024.
13. Thornburg, C.D., Simmons, D.H., von Drygalski, A. Evaluating gene therapy as a potential paradigm shift in treating severe hemophilia. BioDrugs. 2023. DOI: 10.1007/s40259-023-00615-4.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCA/LCD): N/A