Valoctocogene Roxaparvovec
Valoctocogene roxaparvovec is a gene therapy medication used to treat severe hemophilia A in adults without pre-existing antibodies to adeno-associated virus serotype 5 (AAV5). It is administered as a single intravenous infusion and works by delivering a functional copy of the human factor VIII gene, enabling the patient's body to produce its own clotting factor.
Valoctocogene roxaparvovec is a one-time gene therapy for adults with severe hemophilia A that enables endogenous production of factor VIII.
Key facts at a glance
- ATC
- B02BD15
- RxNorm
- 2641610
- Generic
- Valoctocogene Roxaparvovec
- Class
- atc-b02
- Brands
- Roctavian
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks; discuss with healthcare provider
regular fetal monitoring if used
pregnancy
second trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
pregnancy
third trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring
lactation
breastfeedingno adjustment recommended but avoid
breastfeeding not recommended for at least 6 months after infusion due to unknown excretion in breast milk
monitor infant for adverse effects
pediatric_age
children under 12not applicable
Roctavian is not approved for patients under 18 years of age; pediatric use is not recommended
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
geriatric
elderly over 65no specific dosage adjustment
use with caution; limited clinical trial data in patients over 65; assess cardiovascular and hepatic status
monitor liver function tests and cardiovascular status
Use with caution and monitor for adverse effects.
renal_impairment
moderate to severeno specific dosage adjustment in labeling
use with caution; patients with severe renal impairment (eGFR less than 30 mL/min) were excluded from clinical trials; monitor renal function
monitor renal function before and after treatment
Use with caution and monitor for adverse effects.
hepatic_impairment
significant hepatic impairmentavoid
contraindicated in patients with significant hepatic impairment including elevated ALT or AST greater than 1.5x ULN, cirrhosis, or evidence of liver dysfunction; AAV vector targets hepatocytes
monitor liver function tests; liver biopsy may be required
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
Drug class
Gene therapy for hemophilia A
Indication
Therapeutic indication
Primary endpoint achievement
Phase 3 clinical trial results
Administration route
Method of delivery
Treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without pre-existing antibodies to AAV5 and with no history of factor VIII inhibitors.
This AAV5-based gene therapy delivers a functional copy of the human factor VIII (F8) gene directly to liver cells, allowing them to produce and secrete endogenous factor VIII protein, thereby improving clotting capability.
Administered as a single infusion; liver enzyme (transaminase) monitoring is required post-infusion; corticosteroids may be needed to manage liver enzyme elevations; patients should be monitored long-term for factor VIII activity levels and potential hepatocellular effects.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 24, 2026. · Read our editorial policy