Product Specs Roctavian
Manufacturer Name BioMarin
Indications ROCTAVIAN is an adeno-associated virus vector-based gene therapy 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.
Contraindications Contraindicated in individuals with:

  • Active infections, either acute or uncontrolled chronic.
  • Known significant hepatic fibrosis (stage 3 or 4), or cirrhosis.
  • Known hypersensitivity to mannitol.
  • Viral Safety Process
    Product Half-Life Factor VIII mean (SD) activity levels (IU/dL) over time were 84.9 (83.1), 64.7 (64.6), 38.9 (50.7), and 33.8 (47.6) respectively, at 6, 12, 24, and 36 months; measured using one-stage clotting assays (OSA) method.
    Manufacturing Method ROCTAVIAN (valoctocogene roxaparvovec-rvox) is an adenoassociated virus (AAV) vector-based gene therapy product. ROCTAVIAN is replication-incompetent and consists of an AAV serotype 5 capsid containing a DNA sequence encoding the B-domain deleted SQ form of the human coagulation factor VIII. Derived from naturally occurring adeno-associated virus and is produced using Sf9 insect cells and recombinant baculovirus technology.
    Storage Requirements/Shelf Life

    Product as Packaged for Sale:

  • Store frozen at ≤ -60°C (-76°F)
  • Store upright and protect from light in carton until ready to use.
  • During Preparation and Administration:

  • Thaw at room temperature, up to 25°C (77°F).
  • Hold at room temperature for a maximum of 10 hours, including preparation and infusion times.
  • An intact vial (stopper not yet punctured) that has been thawed can be stored refrigerated (2 to 8 °C) for up to 3 days, upright and protected from light.
  • Do not expose to ultraviolet radiation disinfection lamp.
  • Once thawed, DO NOT REFREEZE.
  • Nominal Vial Size & Diluent Volume Each carton contains one single-dose vial with an extractable volume of not less than 8 mL, containing 16 × 1013 vector genomes (vg) or 2 × 1013 vg/mL
    Route of Administration Intravenous Administration

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