HEMGENIX
etranacogene dezaparvovec · KIT · CSL Behring
Hemgenix is a gene therapy medication used to treat severe hemophilia B, a bleeding disorder caused by lack of a clotting protein in the blood. It works by delivering a functional copy of the gene needed to produce this clotting factor.
Key Facts
- Brand Name
- HEMGENIX
- Generic Name
- etranacogene dezaparvovec
- NDC Code (Product)
0053-0260- Manufacturer
- CSL Behring
- Dosage Form
- KIT
- Marketing Status
- Application #
- BLA125772
- Marketing Start
- 11/22/2022
Recall History
No Recall HistorySide Effects Reported to FDA
FDA FAERS database · These are reported events, not confirmed side effects
Full Prescribing Information
Indications & Usage
1 INDICATIONS AND USAGE HEMGENIX is indicated for treatment of adults with 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. HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with 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.
Dosage & Administration
2 DOSAGE AND ADMINISTRATION For single-use intravenous infusion only. ( 2 ) Perform baseline testing to select patients, including testing for Factor IX inhibitor presence and liver health tests. ( 2.1 ) The recommended dose of HEMGENIX is 2 × 10 13 genome copies (gc) per kg of body weight. ( 2.1 ) Administer HEMGENIX as an intravenous infusion after dilution with 0.9% normal saline at a constant infusion rate of 500 ml/hour (8 mL/min). ( 2.1 ) 2.1 Critical Administration-related Information For single-use intravenous infusion only. For patient selection: Perform Factor IX inhibitor titer testing. Do not administer HEMGENIX for patients with positive FIX inhibitors or a prior history for FIX inhibitors. Perform liver health assessments, including: Enzyme testing [alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP) and total bilirubin)], hepatic ultrasound and elastography. In case of radiological liver abnormalities and/or sustained liver enzyme elevations, consider a consultation with hepatologist to assess eligibility for HEMGENIX. Perform laboratory tests to evaluate active hepatitis B and C. Postpone HEMGENIX treatment until patient does…
Contraindications
4 CONTRAINDICATIONS None. None. ( 4 )
Adverse Reactions
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥5%) were elevated ALT, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, malaise and elevated AST. ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact CSL Behring at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice. The safety of HEMGENIX was evaluated in two clinical studies (study 1 enrolled 3 patients and study 2 enrolled 54 patients). Both studies enrolled adult male patients with moderately severe or severe Hemophilia B (N = 57), who received a single intravenous dose of 2 × 10 13 gc/kg body weight of HEMGENIX. Three patients in study 1 and 50 of 54 patients from study 2 completed the study-specific 5-year follow-up period. No serious adverse reactions were reported [see Clinical Studies (14) ] . The most common adverse reactions observed in ≥5% of…
Frequently Asked Questions
What is HEMGENIX used for?
Hemgenix is a gene therapy medication used to treat severe hemophilia B, a bleeding disorder caused by lack of a clotting protein in the blood. It works by delivering a functional copy of the gene needed to produce this clotting factor.
Is HEMGENIX a controlled substance?
HEMGENIX is not classified as a controlled substance by the DEA.
What is the generic name for HEMGENIX?
The generic name for HEMGENIX is etranacogene dezaparvovec. There are no other listed brand versions of etranacogene dezaparvovec.
What is the NDC code for HEMGENIX ?
The NDC (National Drug Code) for HEMGENIX is 0053-0260, listed by CSL Behring.
Not medical advice. Always consult your doctor or pharmacist before making any medication decisions.
Data from openFDA · Public domain (CC0 1.0)