Roctavian

Product Specs	Description
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