Pharmacy Prior Authorization Criteria

Roctavian (valoctocogene roxaparvovec-rvox)

Indications for Prior Authorization

Roctavian (valoctocogene roxaparvovec-rvox)

• For diagnosis of Hemophilia A
  Indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

Criteria

Roctavian

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime
For diagnosis of Hemophilia A

• Diagnosis of severe Hemophilia A
AND
• Factor VIII (FVIII) assay baseline level of less than 1 IU/dL
AND
• Patient is 18 years of age or older
AND
• Patient does not have pre-existing immunity to the adeno-associated virus serotype 5 (AAV5) capsid as detected by the FDA-approved companion diagnostic test [1, A]
AND
• Patient does not have a history of inhibitors based on results from a modified Nijmegen Bethesda assay of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least 1 week apart within the past 12 months [2]
AND
• Treatment logs including both factor infusions and bleeding episodes confirming BOTH of the following [2]:
• Patient has been on prophylactic FVIII replacement therapy for at least 12 months
AND
• Patient has been treated/exposed to FVIII concentrates for a minimum 150 exposure days (EDs) [2, 3, B]
AND
• One of the following:
• Patient does not exhibit significant liver dysfunction as defined by abnormal elevation of ONE of the following [2]:
• alanine transaminase (ALT) greater than 1.25 times the upper limit of normal
• aspartate aminotransferase (AST) greater than 1.25 times the upper limit of normal
• gamma-glutamyl transferase (GGT) greater than 1.25 times the upper limit of normal
• alkaline phosphatase (ALP) greater than 1.25 times the upper limit of normal
• bilirubin greater than 1.25 times the upper limit of normal
• international normalized ratio (INR) greater than or equal to 1.4
OR
• Patient has had a consultation with a hepatologist to assess eligibility for Roctavian
AND
• Patient does not have an active infection or any immunosuppressive disorder [2]
AND
• Patient has never received valoctocogene roxaparvovec treatment in their lifetime
AND
• Prescribed by a hematologist affiliated with a comprehensive hemophilia treatment center (HTC)
AND
• Prescriber attests that the patient has been counseled and has agreed to adhere to post-treatment monitoring and follow-ups with their hematologist and HTC [4]

P & T Revisions

2025-07-14, 2024-12-02, 2023-08-07, 2023-07-20

References

1. Biomarin's Valrox, possible 1st gene therapy for hemophilia A, Under FDA priority review. Hemophilia News Today; February 2020. Available at: https://hemophilianewstoday.com/2020/02/24/biomarins-valrox-possible-first-gene-therapy-hemophila-a-under-fda-priority-review-marketing-application-accepted/
2. Pasi K.J., Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAv5-hFVIII-SQ gene therapy for hemophilia A. New Eng J of Med. 2020;382:29-40.
3. Hermans C, Astermark J, de Moerloose P. Exposure to factor VIII and prediction of inhibitor development: exposure days vs.danger days, or both? J Thromb Haemost 2012;10:2194.
4. Per clinical consult with hematologist, May 29, 2020.
5. Roctavian Prescribing Information. BioMarin Pharmaceutical Inc. Novato, CA. June 2023.

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End Notes

1. About 20% of patients with Hemophilia A in the U.S are estimated to produce antibodies, which could make them ineligible for AAV5-mediated gene therapy. [1]
2. A.B. The incidence rate of inhibitor development in patients with hemophilia A who have been previously treated for at least 150 EDs has been estimated to be approximately 2–5 per 1000 patient-years. [3]

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Revision History

• 2025-07-14: 2025 Annual Review - Program verbiage update with no changes to clinical intent.
• 2024-12-02: 2024 Annual Review
• 2023-08-07: Update criteria to be "Factor VIII (FVIII) assay baseline level of less than 1 IU/dL"
• 2023-07-20: New Program

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