Fidanacogene Elaparvovec
Fidanacogene elaparvovec is a gene therapy product designed to treat hemophilia B, a rare genetic bleeding disorder characterized by deficiency of clotting Factor IX. This one-time treatment works by delivering a functional copy of the Factor IX gene into the patient's liver cells using an adeno-associated virus (AAV) vector, enabling the body to produce its own clotting factor. It is specifically indicated for individuals with moderate to severe hemophilia B.
A gene therapy for hemophilia B that enables the liver to produce its own clotting Factor IX, reducing or eliminating the need for regular factor replacement infusions.
Key facts at a glance
- ATC
- B02BD17
- RxNorm
- 2681011
- Generic
- Fidanacogene Elaparvovec
- Class
- atc-b02
- Brands
- Beqvez
- 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
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2×10^14 vg/kg
- Maximum
- consult physician
- Frequency
- single administration
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
7 groupspregnancy
all trimestersavoid use
Avoid use during pregnancy unless benefits outweigh risks. This is a single-dose gene therapy that may persist in the body long-term.
regular fetal monitoring if used
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
first trimesteravoid if possible
avoid use during first trimester unless benefits outweigh risks
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingno adjustment - avoid use
It is unknown whether fidanacogene elaparvovec is excreted in human breast milk. Breastfeeding is not recommended for 6 months post-infusion due to potential transmission via bodily fluids.
none established
geriatric
elderly 65 plususe with caution - no specific dose adjustment
Limited clinical trial data in patients over 65 years. Assess cardiovascular status, liver function, and overall health before treatment. May have reduced efficacy due to age-related factors.
enhanced liver function monitoring, cardiovascular monitoring
Use with caution and monitor for adverse effects.
pediatric_age
adolescents 12 to 17not recommended - not approved for patients under 18
Not approved for patients under 18 years of age. The FDA approval is for adults 18 years and older with hemophilia B. Consider only in clinical trials with appropriate institutional review board approval.
long-term follow-up per gene therapy registry requirements
Use with caution and monitor for adverse effects.
renal_impairment
severe renal impairmentuse with caution
Patients with severe renal impairment (creatinine clearance <30 mL/min) were excluded from clinical trials. Use only if benefits outweigh risks. Monitor renal function closely.
monitor renal function tests regularly pre and post treatment
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate hepatic impairmentuse with caution - assess liver function
Patients with moderate to severe hepatic impairment (Child-Pugh B or C) were excluded from clinical trials. The liver is the target organ for Factor IX expression. Use only after careful risk-benefit assessment.
comprehensive liver function monitoring including ALT, AST, bilirubin, and liver imaging
Use with caution and monitor for adverse effects.
Statistics and clinical data
Therapy type
Gene therapy for hemophilia B using adeno-associated virus vector
Indication
Treatment of adults with hemophilia B who require routine factor IX prophylaxis
FDA approval year
FDA approval date for Beqvez (fidanacogene elaparvovec)
Manufacturer
Pharmaceutical company responsible for Beqvez
Treatment of moderate to severe hemophilia B (Factor IX deficiency) in adult patients who have circulating anti-AAV antibodies or have unacceptable clinical risk factors for immune tolerance induction to Factor IX.
This AAV-based gene therapy delivers a functional human Factor IX gene to liver cells, allowing them to produce and secrete clotting Factor IX. The goal is to increase systemic Factor IX activity levels to reduce or eliminate the need for prophylactic factor replacement therapy.
As a one-time intravenous infusion, this therapy may cause immune-mediated liver enzyme elevations requiring corticosteroid monitoring. Patients are monitored for Factor IX activity levels and potential hepatocellular injury. Long-term durability of expression and late safety signals continue to be studied in post-marketing surveillance.
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