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.

What patients were included in the HEMGENIX HOPE-B study?
In the HEMGENIX pivotal Phase 3 study, a multinational open-label study:
  • 54 patients received HEMGENIX and completed 12 months of follow-up
  • 53 patients completed 18 months of follow-up and continue to be monitored
Key inclusion criteria for Phase 3 study1:

Male; ≥18 years of age; has congenital hemophilia B classified as moderately severe or severe (factor IX activity ≤2% of normal), and had both >150 previous exposure days to factor IX and received continuous prophylaxis for ≥2 months.

Key exclusion criteria for Phase 3 study1:

Factor IX inhibitors, active hepatitis B or hepatitis C infection, and/or uncontrolled HIV infection—as well as evidence of advanced liver disease.

* The full analysis set (FAS) included 1 patient who died at month 15 post-dose due to unrelated concomitant disease.

How is HEMGENIX dosed?

HEMGENIX is a suspension for intravenous infusion after dilution. It is provided in kits containing 10-48 vials; each kit is ordered based on the patient’s body weight. HEMGENIX has a nominal concentration of 1 x 1013 genome copies (gc)/mL, and each vial contains an extractable volume of not less than 10 mL. The recommended dose of HEMGENIX is 2 x 1013 gc per kg of body weight (or 2 mL per kg body weight) administered as an intravenous infusion after dilution with 0.9% normal saline.

How long does it take to administer HEMGENIX?
HEMGENIX is administered as a one-time-only intravenous infusion—delivered in 1-2 hours in an outpatient setting. No special pretreatment of patients is required.
  • Each dosing kit is personalized to your patient’s weight
  • Delivered as a single dose of 2 mL/kg weight*

*Further dilution with 0.9% normal saline is required.

See which patients could benefit from HEMGENIX Explore sample profiles
Still have questions about HEMGENIX? Get in touch with our team. Connect with us

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

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

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 www.fda.gov/medwatch.