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

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

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%)

Mechanism of action

• adeno-associated virus vector-based gene therapy

More general terms

• gene therapy