• Bleeding disorders

Hemgenix Generic Name & Formulations

General Description

Etranacogene dezaparvovec-drlb 1x10^13 genome copies (gc); per mL; susp for IV infusion after dilution; preservative-free.

Pharmacological Class

Adeno-associated virus vector-based gene therapy.

How Supplied

Customized kit—1 (10–48 vials)


Generic Availability


Mechanism of Action

Hemgenix is an adeno-associated virus vector-based gene therapy designed to deliver a copy of a gene encoding the Padua variant of human coagulation Factor IX (hFIX-Padua). A single intravenous dose of Hemgenix results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.

Hemgenix Indications


Hemophilia B in adults who: currently use Factor IX prophylaxis, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Hemgenix Dosage and Administration


See full labeling. Perform Factor IX inhibitor titer testing and liver assessments (ALT, AST, ALP, bilirubin) prior to treatment. Give as IV infusion at a rate of 500mL/hr (8mL/min). 2×1013gc/kg (2mL/kg). 


Not established. 

Hemgenix Contraindications

Not Applicable

Hemgenix Boxed Warnings

Not Applicable

Hemgenix Warnings/Precautions


Monitor for infusion reactions during and for ≥3hrs after completion; manage appropriately if occurs. Hepatotoxicity. Monitor transaminase levels once weekly for 3 months after administration; if liver enzyme elevations developed, continue to monitor until return to baseline. Preexisting risk factors for hepatocellular carcinoma (eg, cirrhosis, advanced hepatic fibrosis, hepatitis B or C, non-alcoholic fatty liver disease, chronic alcohol consumption, non-alcoholic steatohepatitis, and advanced age), perform regular (eg, annual) liver ultrasound and alpha-fetoprotein testing after administration. Monitor for Factor IX activity and appropriate testing for development of Factor IX inhibitors. Advanced hepatic impairment (including cirrhosis, advanced liver fibrosis, or uncontrolled Hepatitis B and C), severe renal impairment, ESRD: not studied. Not intended for use in women.

Hemgenix Pharmacokinetics

See Literature

Hemgenix Interactions

Not Applicable

Hemgenix Adverse Reactions

Adverse Reactions

Elevated ALT/AST, blood creatine kinase elevations, headache, flu-like symptoms, infusion-related reactions, fatigue, malaise.

Hemgenix Clinical Trials

See Literature

Hemgenix Note

Not Applicable

Hemgenix Patient Counseling

See Literature