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Roctavian® (valoctocogene roxaparvovec-rvox)

Policy Number: PH-0718

Intravenous

Last Review Date: 06/04/2024

Date of Origin: 08/08/2023

Dates Reviewed: 08/2023, 06/2024

FOR PEEHIP Members Only -Coverage excludes the provider-administered medication(s) outlined in this drug policy from being accessed through a specialty pharmacy. It must be obtained through buy and bill.

  1. Length of Authorization

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

  1. Dosing Limits

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

  • Roctavian 2 x 1013 vg/mL single-dose vial: 44 vials one time only

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

  • 352 billable units (352 mL) one time only
  1. Initial Approval Criteria 1-15

Submission of medical records (chart notes) related to the medical necessity criteria is REQUIRED on all requests for authorizations. Records will be reviewed at the time of submission. Please provide documentation related to diagnosis, step therapy, and clinical markers (i.e., genetic, and mutational testing) supporting initiation when applicable.  Please provide documentation via direct upload through the PA web portal or by fax.

Coverage is provided in the following conditions:

Hemophilia A (Congenital Factor VIII Deficiency) † Ф

  • Patient is at least 18 years of age; AND
  • Patient has a diagnosis of severe hemophilia A (congenital factor VIII deficiency) as confirmed by a factor VIII activity level < 1 IU/dL; AND
  • Evidence of any bleeding disorder NOT related to hemophilia A has been ruled out; AND
  • Patient is on a stable dose of regularly administered exogenous factor VIII for the prevention and control of bleeding episodes; AND
  • Patient does not have an active infection, either acute (such as acute respiratory infections or acute hepatitis) or uncontrolled chronic (such as chronic active hepatitis B); AND
  • Patient is up to date with vaccinations prior to infusion and will avoid live vaccines while on immunosuppressive therapies; AND
  • Patient does not have significant hepatic fibrosis (stage 3 or 4) or cirrhosis; AND
  • Patient does not have a known hypersensitivity to mannitol: AND
  • Patient has not received prior hemophilia AAV-vector–based gene therapy; AND
  • Patient is adeno-associated virus serotype 5 (AAV5) antibody negative as determined by an FDA-approved or CLIA-compliant testv; AND
  • Patient has been tested and found negative for active factor VIII inhibitors (i.e., results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least one week apart within the past 12 months) and is not receiving a bypassing agent (e.g., Feiba, NovoSeven RT, SevenFact, etc.); AND
  • Patient will have post-administration monitoring of serum ALT levels performed according to the monitoring schedule outlined in the product labeling with corticosteroids (or other immunosuppressive therapy) administered in response to elevations; AND
  • Patients with preexisting risk factors for hepatocellular carcinoma [e.g., patients with hepatitis C or B, non-alcoholic fatty liver disease (NAFLD), chronic alcohol consumption, non-alcoholic steatohepatitis (NASH), and advanced age] will have regular (e.g., annually) liver ultrasounds performed and will be tested for alpha-fetoprotein (AFP) elevations following administration; AND
  • Patient will have factor VIII activity monitored according to the monitoring schedule outlined in the product labeling; AND
    • Patients with factor VIII activity levels >5 IU/dL will discontinue routine prophylactic exogenous factor VIII; OR
    • If the factor VIII activity levels decrease and/or if bleeding is not controlled, the patient will be assessed for the presence of factor VIII inhibitors and the need for hemostatic prophylaxis

Notes:

  • It may take several weeks after valoctocogene roxaparvovec infusion before valoctocogene roxaparvovec-derived factor VIII activity rises to a level sufficient for prevention of spontaneous bleeding episodes. Therefore, continued routine prophylaxis support with exogenous factor VIII or other hemostatic products used in the management of hemophilia A may be needed during the first few weeks after valoctocogene roxaparvovec infusion.
  • Exogenous factor VIII or other hemostatic products may continue to be required in the case of surgery, invasive procedures, trauma, or bleeds in the event that valoctocogene roxaparvovec-derived factor VIII activity is deemed insufficient for adequate hemostasis in such situations.
  • Use of exogenous factor VIII products before and after valoctocogene roxaparvovec administration may impede assessment of valoctocogene roxaparvovec-derived factor VIII activity.

v If confirmed using an immunotherapy assay-http://www.fda.gov/companiondiagnostics

FDA Approved Indication(s); Compendia Recommended Indication(s); Ф Orphan Drug

  1. Renewal Criteria 1

Coverage cannot be renewed.

  1. Dosage/Administration 1

Indication

Dose

Hemophilia A (Congenital Factor VIII Deficiency)

The recommended dose of Roctavian is 6 × 1013 vector genomes per kilogram (vg/kg) body

weight, administered as a single intravenous infusion.

Calculating Dose in Milliliters (mL) and Number of Vials Required

Patient dose volume in mL:

  • Body weight in kg multiplied by 3 = dose in mL.
  • The multiplication factor 3 represents the per kilogram dose (6 × 1013 vg/kg) divided by the amount of vector genomes per mL of suspension (2 × 1013 vg/mL).

Number of vials to be thawed:

  • Patient dose volume (mL) divided by 8 = number of vials to be thawed (round up to next whole number of vials).
  • The division factor 8 represents the minimum volume extractable from a vial (8 mL).
  • Roctavian is administered using an infusion pump at a rate of 1 mL/min, which can be increased every 30 minutes by 1 mL/min up to a maximum rate of 4 mL/min.
  • Do not expose Roctavian to the light of an ultraviolet radiation disinfection lamp.
  • Prepare using aseptic technique. Wear gloves and safety glasses during preparation and administration.
  • Treat spills with a virucidal agent with proven activity against non-enveloped viruses and blot using absorbent materials.
  • Dispose unused medicinal product and materials that may have come in contact with Roctavian in accordance with the local biosafety guidelines.
  • Thaw at room temperature. Do not thaw or warm vials any other way. Thawing time is approximately 2 hours. Thawed suspension can be held at room temperature, up to 25°C (77°F), for a maximum of 10 hours including hold time in intact vial, preparation time into the syringes, and duration of infusion.
  • DO NOT administer as an intravenous push or bolus.
  • DO NOT infuse in the same intravenous line with any other products.
  • DO NOT use a central line or port.
  1. Billing Code/Availability Information

HCPCS code:

  • J1412 – Injection, valoctocogene roxaparvovec-rvox, per mL, containing nominal 2 × 1013 vector genomes; 1 billable unit = 1 mL, containing nominal 2 × 1013 vector genomes

NDC:

  • Roctavian 2 × 1013 vector genomes (vg) per mL – 8 mL single dose vial: 68135-0927-xx
  1. References
  1. Roctavian [package insert]. Novato, CA; BioMarin Pharm., LLC., June 2023. Accessed May 2024.
  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. 3rd 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); April 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;10:615621.
  10. Ozelo MC, et al. Valoctocogene roxaparvovec gene therapy for hemophilia A. N Engl J Med. 2022;386(11):1013-1025. doi:10.1056/NEJMoa2113708.
  11. Rind DM, et al. Valoctocogene roxaparvovec and emicizumab for hemophilia A without inhibitors: effectiveness and value; final report. Institute for Clinical and Economic Review. Published November 20, 2020. https://icer.org/wp-content/uploads/2020/10/ICER_Hemophilia-A_Final-Report_112020.pdf
  12. Mahlangu J, Kaczmarek R, von Drygalski A, et al. Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A. N Engl J Med. 2023;388(8):694-705. doi: 10.1056/NEJMoa2211075.
  13. Madan B, Ozelo MC, Raheja P, et al. Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A. J Thromb Haemost. 2024 Apr 11:S1538-7836(24)00184-3. doi: 10.1016/j.jtha.2024.04.001.
  14. MASAC Recommendations on Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia. National Hemophilia Foundation. MASAC Document # 282 (Replaces Document #277). 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.
  15. 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

ICD-10

ICD-10 Description

D66

Hereditary factor VIII deficiency

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

Medicare Part B Administrative Contractor (MAC) Jurisdictions

Jurisdiction

Applicable State/US Territory

Contractor

E (1)

CA, HI, NV, AS, GU, CNMI

Noridian Healthcare Solutions, LLC

F (2 & 3)

AK, WA, OR, ID, ND, SD, MT, WY, UT, AZ

Noridian Healthcare Solutions, LLC

5

KS, NE, IA, MO

Wisconsin Physicians Service Insurance Corp (WPS)

6

MN, WI, IL

National Government Services, Inc. (NGS)

H (4 & 7)

LA, AR, MS, TX, OK, CO, NM

Novitas Solutions, Inc.

8

MI, IN

Wisconsin Physicians Service Insurance Corp (WPS)

N (9)

FL, PR, VI

First Coast Service Options, Inc.

J (10)

TN, GA, AL

Palmetto GBA

M (11)

NC, SC, WV, VA (excluding below)

Palmetto GBA

L (12)

DE, MD, PA, NJ, DC (includes Arlington & Fairfax counties and the city of Alexandria in VA)

Novitas Solutions, Inc.

K (13 & 14)

NY, CT, MA, RI, VT, ME, NH

National Government Services, Inc. (NGS)

15

KY, OH

CGS Administrators, LLC