Primary navigation

    Roctavian™

    Drug - Roctavian™ (valoctocogene roxaparvovec-rvox) [BioMarin Pharmaceuticals, Inc.]

    March 2025

    Therapeutic Area - Hemophilia Treatment

    Approval criteria

    • Patient is at least 18 years of age; AND
    • Patient has severe hemophilia A (congenital factor VIII deficiency) diagnosed by a factor VIII activity level < 1 IU/dL (in the absence of exogenous factor VIII); 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
    • Must not be administered concurrently with 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 test; 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); AND 
    • Post administration monitoring of patient serum ALT levels will be 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 Factor VIII activity will be monitored periodically; AND 
      • Patients with factor VIII activity levels >5 IU/dL should discontinue routine prophylactic exogenous factor VIII; OR 
      • If Factor VIII activity levels decrease and/or if bleeding is not controlled, assess presence of factor VIII inhibitors and assess the need for hemostatic prophylaxis

    Quantity limits

    • Patient’s body weight and the corresponding requested number of vials need to be submitted at the time of request
    • Coverage will be provided for one dose to eligible patient meeting all approval criteria and may not be renewed

    Billing for Roctavian

    Roctavian must be billed as a medical claim.  

    Questions

    Provider Call Center (844) 575-7887

    back to top