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Key Publications


Gene therapy with etranacogene dezaparvovec for hemophilia B
Pipe SW, Leebeek FWG, Recht M, et al. N Engl J Med. 2023
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Stable and durable factor IX levels in hemophilia B patients over 3 years post etranacogene dezaparvovec gene therapy
von Drygalski A, Gomez E, Giermasz A, et al. Blood Adv 2022
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Adults With Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection After 24 Months’ Follow-up

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Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals With and Without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <1:700) With Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B

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Please see full prescribing information for HEMGENIX

Improvements in health-related quality of life in adults with severe or moderately severe hemophilia B after receiving etranacogene dezaparvovec gene therapy

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Please see full prescribing information for HEMGENIX

Analysis of elevated alanine transaminase in HOPE B, a Phase 3 recombinant adeno associated viral 5 gene therapy trial in people with haemophilia B

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Please see full prescribing information for HEMGENIX

Durability of Factor IX Activity and Bleeding Rate in People With Severe or Moderately Severe Hemophilia B After 5 Years of Follow-Up in the Phase 1/2 Study of AMT-060, and After 3 Years of Follow-Up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)

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Please see full prescribing information for HEMGENIX

Multiple-year durability data from a Phase 2b trial of gene therapy with etranacogene dezaparvovec in people with hemophilia B

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Please see full prescribing information for HEMGENIX

Important Safety Information for HEMGENIX

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.

Indication for HEMGENIX

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

For U.S. Healthcare Professionals only
For U.S. Healthcare Professionals only

The purpose of this CSL Behring Medical Affairs website is to support Healthcare Professionals with scientific information. This website is also a channel for U.S. Healthcare Professionals to submit questions or connect with CSL Behring U.S. Healthcare Professionals. The information provided is for educational purposes only and is not intended to promote any products. By continuing to use this site you are acknowledging that you are a US Healthcare Professional

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