Formula & Concentration
- ROCTAVIAN (valoctocogene roxaparvovec-rvox) suspension for intravenous infusion
- ROCTAVIAN has a nominal concentration of 2 × 1013 vg valoctocogene roxaparvovec-rvox per mL, each vial contains an extractable volume of not less than 8 mL (16 x 1013 vg)
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.
- This is a one-time therapy
Shelf Life and Storage
- Product as Packaged for Sale
- Transport frozen at ≤ -60°C (-76°F)
- Store upright at ≤ -60°C (-76°F)
- Store ROCTAVIAN vial in carton until ready to use.
- Protect ROCTAVIAN from light.
- During Preparation and Administration
- Thaw at room temperature, up to 25°C (77°F).
- After thawing, ROCTAVIAN can be held at room temperature for a maximum of 10 hours, including preparation and infusion times.
- If necessary, 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 (e.g., in the original carton).
- Do not expose ROCTAVIAN to the light of an ultraviolet radiation disinfection lamp.
- Once thawed, DO NOT REFREEZE.
- Treat spills of ROCTAVIAN with a virucidal agent with proven activity against non-enveloped viruses and blot using absorbent materials.
- Dispose of unused product and disposable materials that may have come in contact with ROCTAVIAN in accordance with local guidance for pharmaceutical waste.
- Dating Period
- The dating period for valoctocogene roxaparvovec-rvox shall be 36 months from the date of manufacture when stored at -60°C. The date of manufacture shall be defined as the date of final sterile filtration of the formulated drug product. Following the final sterile filtration, no reprocessing/reworking is allowed without prior approval from the Agency. The dating period for your drug substance shall be 12 months when stored at -60°C or 21 days when stored at 2-8° We have approved the stability protocol in your license application for the purpose of extending the expiration dating period of your drug substance and drug product under 21 CFR 601.12