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etranacogene dezaparvovec-drlb (Hemgenix)
Indications:
- Congenital hemophilia B
- defects in the gene encoding factor IX
Dosage:
- 2 x 10E13 genome copies/kg body weight
- 2 x patient weight in Kg = dose in mL
- dose in vials = dose in mL/10 rounded up
- for 72 kg patient 144 mL, 15 vials
- administer as anintravenous infusion after dilution with 0.9% normal saline
- maintain constant infusion rate of 8 mL/min
* Hemgenix can be administered only once [2]
provided in kits containing 10 to 48 single-use vials, each kit constituting a dosage unit based on the patient's weight
10 mL etranacogene dezaparvovec-drlb 10E13 VECTOR-GENOMES/mL Injection
Monitor:
- serum ALT* & serum AST* once per week for 3 months after administration
- annual liver ultrasound & serum alpha-fetoprotein testing for patients with pre-exsiting risk factors for hepatocellular carcinoma
* condider glucocorticoid for elevated serum ALT [2]
Adverse effects:
- hepatotoxicity
- hepatocellular carcinoma for patients with pre-exsiting risk factors
- elevated serum ALT (24%), serum AST (42%), serum creatine kinase (42%)
- headache (18%), flu-like symptoms (14%), fatigue (12%), malaise (12%), nausea (7%)
- infusion-related reactions (13%)
- hypersensitivity (4%) [2]
Mechanism of action:
- adeno-associated virus vector-based gene therapy [2]
General
gene therapy
References
- Shah J, Kim h, Sivamurthy K et al
Comprehensive analysis and prediction of long-term durability of factor IX activity
following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B.
Curr Med Res Opin. 2023 Feb;39(2):227-237.
PMID: 36285399
https://www.tandfonline.com/doi/full/10.1080/03007995.2022.2133492
- HIGHLIGHTS OF PRESCRIBING INFORMATION
Hemgenix (etranacogene dezaparvovec-drlb) suspension, for intravenous infusion
https://labeling.cslbehring.com/PI/US/Hemgenix/EN/Hemgenix-Prescribing-Information.pdf