Pharmacy Prior Authorization Criteria

Hemophilia B Gene Therapies - PA, NF

Indications for Prior Authorization

Hemgenix (etranacogene dezaparvovec-drlb)

• For diagnosis of Hemophilia B
  Indicated for treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Beqvez (fidanacogene elaparvovec-dzkt)

• For diagnosis of Hemophilia B
  Indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and, do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. Select patients for therapy based on an FDA-approved companion diagnostic for Beqvez

Criteria

Hemgenix

Prior Authorization, Non Formulary

Length of Approval: 1 Time Authorization in Lifetime*

• Submission of medical records (e.g., chart notes) confirming diagnosis of Hemophilia B (congenital Factor IX deficiency)
AND
• Submission of medical records (e.g., chart notes) documenting one of the following:
• Both of the following:
• Diagnosis of severe hemophilia B
• Documentation of endogenous Factor IX levels less than 1% of normal Factor IX (< 0.01 IU/mL)
OR
• All of the following:
• Diagnosis of moderately severe hemophilia B
• Documentation of endogenous Factor IX levels greater than or equal to 1% to less than or equal to 2% (greater than or equal to 0.01 IU/mL to less than or equal to 0.02 IU/mL)
• Patient has current or historical life-threatening hemorrhage or repeated, serious spontaneous bleeding episodes
AND
• Paid claims or submission of medical records (e.g., chart notes) confirming that the patient is currently using Factor IX prophylaxis therapy (e.g., BeneFIX, Ixinity, Rixubis, etc.) and will discontinue treatment after stable on Hemgenix therapy
AND
• Patient has greater than 150 previous exposure days of treatment with a Factor IX agent
AND
• Patient is 18 years of age or older
AND
• Submission of medical records (e.g., chart notes) documenting that the patient does not have any of the following:
• Positive human immunodeficiency virus (HIV) test at screening that is not controlled with anti-viral therapy
• Active infection with hepatitis B or C virus
• Currently on antiviral treatment for hepatitis B or C
• Positive Factor IX inhibitor titer test prior to therapy
• History of Factor IX inhibitor
• Anti-AAV antibody (e.g., AAV-5) titers exceeding 1:678
AND
• Both of the following:
• Submission of medical records (e.g., chart notes) documenting that the following laboratory values have been checked prior to therapy and are less than two times the upper limit of normal and provider attests that these laboratory values will be monitored after administration according to the FDA-approved recommendations in the labeling:
• Alanine aminotransferase (ALT)
• Alkaline phosphatase (ALP)
• Aspartate aminotransferase (AST)
• Total bilirubin
AND
• Submission of medical records (e.g., chart notes) documenting results of the following baseline tests and provider attests there will be ongoing monitoring according to the recommendations in the FDA labeling:
• Factor IX activity (i.e., Exogenous Factor IX and Human Factor IX inhibitors)
AND
• In patients with preexisting risk factors for hepatocellular carcinoma (e.g., in patients with cirrhosis, advanced hepatic fibrosis, hepatitis B or C, non-alcoholic fatty liver disease (NAFLD), chronic alcohol consumption, non-alcoholic steatohepatitis (NASH), and advanced age), both of the following:
• Alpha-fetoprotein (AFP) level
• Abdominal ultrasound
AND
• Submission of medical records (e.g., chart notes) documenting that hepatic ultrasound and elastography have been completed prior to therapy
AND
• Prescribed by a hematologist at a Hemophilia Treatment Center (HTC)
AND
• Patient has never received any previous Hemophilia B gene therapy treatment in their lifetime (e.g., Hemgenix, Beqvez)

Beqvez

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime*

• Submisson of medical records (e.g., chart notes) confirming diagnosis of Hemophilia B (congenital Factor IX deficiency)
AND
• Submission of medical records (e.g., chart notes) confirming one of the following:
• Both of the following:
• Diagnosis of severe hemophilia B
• Documentation of endogenous Factor IX levels less than 1% of normal Factor IX (< 0.01 IU/mL)
OR
• All of the following:
• Diagnosis of moderately severe hemophilia B
• Documentation of endogenous Factor IX levels greater than or equal to 1% to less than or equal to 2% (greater than or equal to 0.01 IU/mL to less than or equal to 0.02 IU/mL)
• Patient has current or historical life-threatening hemorrhage or repeated, serious spontaneous bleeding episodes
AND
• Paid claims or submission of medical records (e.g., chart notes) confirming patient is currently using Factor IX prophylaxis therapy (e.g., BeneFIX, Ixinity, Rixubis, etc.) and will discontinue treatment after stable on Beqvez therapy
AND
• Both of the following:
• Patient has been on prophylactic Factor IX replacement therapy for at least 6 months
AND
• Patient has greater than 50 previous exposure days of treatment with a Factor IX agent
AND
• Patient is 18 years of age or older
AND
• Submission of medical records (e.g., chart notes) confirming patient does not have any of the following:
• Positive human immunodeficiency virus (HIV) test at screening that is not controlled with anti-viral therapy
• Active infection with hepatitis B or C virus
• Currently on antiviral treatment for hepatitis B or C
• Positive Factor IX inhibitor titer test prior to therapy
• History of Factor IX inhibitor
• Anti-AAVRh74var neutralizing antibodies (nAB)
AND
• Submission of medical records (e.g., chart notes) documenting that the following laboratory values have been checked prior to therapy and are less than two times the upper limit of normal and provider attests that these laboratory values will be monitored after administration according to the FDA-approved recommendations in the labeling:
• Alanine aminotransferase (ALT)
• Alkaline phosphatase (ALP)
• Aspartate aminotransferase (AST)
• Total bilirubin
AND
• Submission of medical records (e.g., chart notes) confirming provider attests that hepatic ultrasound and elastography have been completed prior to therapy
AND
• Prescribed by a hematologist at a Hemophilia Treatment Center (HTC)
AND
• Patient has never received any previous Hemophilia B gene therapy treatment in their lifetime (e.g., Hemgenix, Beqvez)

P & T Revisions

2026-02-10, 2025-01-24, 2024-06-28, 2024-01-10, 2023-01-31

References

1. Hemgenix Prescribing Information. CSL Behring LLC. King of Prussia, PA. January 2025.
2. CSL Behring. Phase III, Open-label, Single-dose, Multi-center, Multinational Trial Investigating a Serotype 5 Adeno-associated Viral Vector Containing the Padua Variant of a Codon-optimized Human Factor IX Gene (AAV5-hFIXco-Padua, AMT-061) Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B. clinicaltrials.gov. Published September 26, 2022. Accessed January 11, 2023. https://clinicaltrials.gov/ct2/show/study/NCT03569891
3. Beqvez Prescribing Information. Pfizer Inc. New York, NY. April 2024.

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Revision History

• 2026-02-10: 2026 Annual Review - updated guideline
• 2025-01-24: 2025 UM Annual Review. No changes
• 2024-06-28: Addition of Beqvez to guideline and updated Hemgenix criteria to state "Patient has never received any previous Hemophilia B gene therapy treatment in their lifetime (e.g., Hemgenix, Beqvez)"
• 2024-01-10: 2024 UM Annual Review. No criteria changes. Background updates
• 2023-01-31: New Program

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