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HEMGENIX®-logo-bg HEMGENIX® (etranacogene dezaparvovec)

Ongoing Multinational Open-Label Phase 3 Study

Pivotal HOPE-B Study Design

Timeline of clinical trial data collection duration Timeline of clinical trial data collection duration
Timeline of clinical trial data collection duration

Primary endpoint

To demonstrate non-inferiority of annualized bleeding rate (ABR) during months 7–18 after HEMGENIX treatment compared with ABR during the lead-in period.

Key secondary endpoints

  • Factor IX activity at 6, 12, and 18 months after dosing
  • Discontinuation of previous continuous routine prophylaxis

Preexisting anti-AAV5 NAbs were assessed but not used as an exclusion criterion, and no prophylactic immunosuppression was required2

Post-treatment follow-up

  • 53 patients completed 18 months of follow-up and continue to be monitored for 5 years
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Administration instructions

Reference: 1. Miesbach W, Frank WG, Leebeek FWG, Recht M, et al; for the HOPE-B Investigators. Final analysis from the pivotal phase 3 HOPE-B gene therapy trial: stable steady-state efficacy and safety of etranacogene dezaparvovec in adults with severe or moderately severe haemophilia B. Presented at: 15th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2022); February 2-4, 2022; Virtual.

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Warning and Precautions

Infusion Reactions

Infusion reactions, including hypersensitivity reactions and anaphylaxis, may occur. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or interrupt administration. Re-start administration at a slower infusion once resolved.

Hepatotoxicity/Hepatocellular Carcinoma

Post-dose, monitor for elevated transaminase levels. Consider corticosteroid treatment should elevations occur. The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. For patients with preexisting risk factors for hepatocellular carcinogenicity, perform regular (eg, annual) abdominal ultrasound and alpha-fetoprotein testing following administration.

Immune-mediated neutralization of the AAV5 vector capsid

Preexisting neutralizing anti-AAV antibodies may impede transgene expression at desired levels.

Monitoring Laboratory Tests

In addition to monitoring liver function, monitor for Factor IX activity and Factor IX inhibitors after administration.

Adverse Reactions

The most common adverse reactions (incidence ≥5%) were elevated ALT, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, nausea, malaise, and elevated AST.


HEMGENIX®, etranacogene dezaparvovec-drlb, 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.

HEMGENIX is for single use intravenous infusion only.

Contraindications: None.

Please see full prescribing information for HEMGENIX.

To report SUSPECTED ADVERSE REACTIONS, contact the CSL Behring Pharmacovigilance Department at 1-866-915-6958 or FDA at 1-800-FDA-1088 or