Etranacogene Dezaparvovec
Etranacogene dezaparvovec (HEMGENIX) is an innovative gene therapy for adults with hemophilia B who currently use Factor IX prophylaxis therapy. It uses an adeno-associated virus serotype 5 (AAV5) vector to deliver a functional copy of the Factor IX gene, specifically the highly active Padua variant, directly into the patient's liver cells.
HEMGENIX is a one-time gene therapy that enables the body to produce its own clotting Factor IX to reduce bleeding episodes in hemophilia B patients.
Key facts at a glance
- ATC
- B02BD16
- RxNorm
- 2626727
- Generic
- Etranacogene Dezaparvovec
- Class
- atc-b02
- Brands
- Hemgenix
- 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
- 2x10^14 gc/kg (maximum 6x10^14 gc)
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2x10^14 gc/kg
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2x10^14 gc/kg (maximum 6x10^14 gc)
- Maximum
- consult physician
- Frequency
- single dose
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
7 groupspregnancy
generalavoid use
avoid use during pregnancy; patients of reproductive potential should use effective contraception for at least 12 months after administration
regular fetal monitoring if pregnancy occurs
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
generalavoid if possible
it is unknown if etranacogene dezaparvovec is excreted in human milk; breastfeeding should be avoided for at least 12 months after administration
monitor infant if breastfeeding continues
pediatric_age
pediatricnot indicated
not approved for use in pediatric patients; indicated only for adults 18 years and older with hemophilia B
not applicable
Use of this medication in this population requires careful consideration of risks and benefits.
geriatric
elderlyno specific dose adjustment required
use with caution; clinical trials included limited patients over 65 years; monitor liver function and infusion-related reactions
regular monitoring of liver enzymes and infusion-related adverse events
Use with caution and monitor for adverse effects.
renal_impairment
severe renal impairmentuse with caution
patients with severe renal impairment were excluded from clinical trials; use only if benefits outweigh risks; monitor renal function
monitor renal function before and after treatment
Use with caution and monitor for adverse effects.
hepatic_impairment
hepatic impairmentdo not administer
contraindicated in patients with pre-existing liver disease including cirrhosis, hepatic fibrosis, or elevated liver enzymes; active infections or conditions that may affect liver function
comprehensive liver assessment including elastography and biopsy if indicated before treatment
Use of this medication in this population requires careful consideration of risks and benefits.
alcohol_users
alcohol usersavoid alcohol consumption
avoid alcohol consumption for at least 12 months after treatment; alcohol may increase liver enzyme elevations and hepatotoxicity risk
monitor liver enzymes regularly; counsel on complete abstinence from alcohol
Use of this medication in this population requires careful consideration of risks and benefits.
Statistics and clinical data
Drug class
AAV vector-based gene therapy for hemophilia B
Indication
FDA-approved indication for treatment of adults with hemophilia B
FDA approval year
First FDA-approved gene therapy for hemophilia B
Administration route
Single intravenous infusion as one-time treatment
Treatment of adults with moderate to severe hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated serious spontaneous bleeding episodes.
As an AAV5-based gene therapy, HEMGENIX delivers a functional gene encoding the Padua variant of human Factor IX directly to liver cells via single intravenous infusion. These cells then produce the clotting factor, increasing circulating Factor IX activity levels and reducing the risk of bleeding.
Administered as a single one-time intravenous infusion only. Requires monitoring of liver enzyme levels (ALT) post-infusion, as elevated levels may require corticosteroids. Does not cure hemophilia B; long-term durability of effect continues to be studied. May cause infusion reactions. Patients should not donate blood, organs, tissues, or cells.
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 July 26, 2026. · Read our editorial policy