You are now leaving the current website.

The site you are accessing is maintained by a third party over whom CSL Behring has no control. CSL Behring does not review, approve, or necessarily endorse viewpoints, inferences, or conclusions stated in or implied by the content of this site. CSL Behring is not responsible for third-party content or the consequences of your use thereof.

Do you want to continue?

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

FREQUENTLY ASKED QUESTIONS

HEMGENIX is the first‑ever gene therapy for hemophilia B, and as you discuss it with your patients, important questions can arise. Here, you can find answers to some of these common questions about treatment with HEMGENIX.

See which patients could benefit from HEMGENIX Explore sample profiles
Still have questions about HEMGENIX? Get in touch with our team. Connect with us
Amelia
Virtual Assistant
Hello, I am Amelia. How can I help you today? If this is a medical emergency, please call 911 or report to your local emergency room.
toggle chat overlay toggle chat overlay

References: 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. 2. Pipe S, Leebeek FWG, Ferreira V, Sawyer EK, Pasi J. Clinical considerations for capsid choice in the development of liver-targeted AAV-based gene transfer. Mol Ther Methods Clin Dev. 2019;15:170-178. doi:10.1016/j.omtm.2019.08.015 3. Vance MA, Mitchell A, Samulski RJ. AAV biology, infectivity and therapeutic use from bench to clinic. In: Hashad D, ed. Gene Therapy: Principles and Challenges [Internet]. IntechOpen; 2015. Accessed October 13, 2022. doi: 10.5772/61988 4. Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407-414. doi:10.1182/blood-2018-07-820720 5. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021 Feb 8;6(1):53. doi:10.1038/s41392-021-00487-6 6. Arruda VR, Doshi BS. Gene therapy for hemophilia: facts and quandaries in the 21st century. Mediterr J Hematol Infect Dis. 2020;12(1):e2020069. doi:10.4084/MJHID.2020.069

Top of Page

IMPORTANT SAFETY INFORMATION

Patient Selection

Perform baseline testing to select patients, including titer testing for Factor IX (FIX) inhibitor presence. Do not administer HEMGENIX®, etranacogene dezaparvovec-drlb, to patients with FIX inhibitors or a history of FIX inhibitors. Perform liver health assessments, consulting with a hepatologist if needed. Also perform laboratory tests to evaluate hepatitis B and C, and postpone treatment if patient has active infection, as this may reduce the efficacy of HEMGENIX and/or increase the risk of adverse reactions.

Warnings and Precautions

Hypersensitivity and Infusion Reactions

Infusion reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or interrupt administration. When symptoms have resolved, restart administration at a slower infusion rate.

Hepatotoxicity/Hepatocellular Carcinogenicity

Hepatotoxicity with elevated liver transaminase has occurred after HEMGENIX treatment. Monitor ALT levels once per week for 3 months and thereafter monthly up to 1 year after administration. Consider corticosteroid treatment should elevations occur and as clinically indicated.

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, consider liver ultrasound and alpha-fetoprotein testing following administration, and monitor for hepatocellular carcinomas for five years following administration of HEMGENIX.

Immune-Mediated Neutralization of the AAV5 vector capsid

Preexisting neutralizing anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with HEMGENIX, all patients developed neutralizing anti-AAV5 antibodies to AAV5 vector capsid.

Monitoring Laboratory Tests

Monitor patients regularly for FIX activity (eg, weekly for 3 months), especially when exogenous FIX is administered, as it may take several weeks following HEMGENIX administration before hemostatic control becomes apparent. Hemostatic support may be needed for some patients. Monitor patients through appropriate clinical observations and laboratory tests for the development of inhibitors to FIX.

Adverse Reactions

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

Indication

HEMGENIX is 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.

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 www.fda.gov/medwatch.