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HEMGENIX®-logo-bg HEMGENIX® (etranacogene dezaparvovec)
37%

Mean Factor IX Activity

sustained at 2 years

Mean factor IX activity levels (N=54)

Infographic of factor IX activity levels with HEMGENIX

Click to enlarge

Infographic of factor IX activity levels with HEMGENIX

*aPTT, activated partial thromboplastin time; FIX, factor IX.

† The full analysis set included 54 patients dosed. Uncontaminated factor IX activity values exclude measurements within five half-lives of factor IX replacement therapy. Contaminated and missing values are not shown here. Specifically, the number of subjects excluded for contamination with factor IX replacement therapy at month 6, month 12, month 18, and month 24, were 3, 3, 3, 2, respectively.

annualized bleed rate (ABR) with down arrow

Significant reduction in annual bleed rate (ABR)

vs. routine factor IX prophylaxis*

HEMGENIX provides significant bleed protection versus factor IX prophylaxis

Reduction in ABR (N=54)

Infographic of annualized bleed rates on prophy vs HEMGENIX

‡ An ABR ratio of 0.46 (95% CI: 0.26, 0.81).

A one-time infusion of HEMGENIX also showed:

  • 63% (34/54) of patients reported zero bleeds in the 7-18 months following treatment
94%

Of patients discontinued

Factor IX prophylaxis

and remained prophylaxis-free†

With Hemgenix,

Most patients eliminated routine factor IX prophylaxis infusions

Pie chart filled 94%
Man standing with hands clasped

Patient portrayal

Discontinued Routine Factor IX Prophylaxis

AND REMAINED PROPHYLAXIS-FREE

§ Two patients were not able to stop routine prophylaxis. During months 7–18, an additional patient received prophylaxis during days 396–534 (approximately 20 weeks).

*The annualized bleed rate (ABR) for all bleeds decreased from an average of 4.1 for prophylaxis during the lead-in period to 1.9 in months 7–18 after treatment, an ABR ratio of 0.46 (95% CI: 0.26, 0.81).

† Two patients were not able to stop routine prophylaxis. During months 7–18, an additional patient received prophylaxis during days 396–534 (approximately 20 weeks).

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

Click to enlarge

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

Key inclusion criteria1

  • Male
  • ≥18 years of age
  • Living with moderately severe or severe hemophilia B
  • Factor IX activity ≤2% of normal
  • Had >150 previous exposure days to factor IX and had received continuous prophylaxis for ≥2 months

Key exclusion criteria1

  • Factors that might affect the evaluation of the efficacy or safety of HEMGENIX, including:
    • Factor IX inhibitors
    • Uncontrolled HIV infection
    • Active hepatitis B or hepatitis C infection
    • Evidence of advanced hepatic impairment

Post-treatment follow-up

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

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

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Learn how to store, prep, and infuse HEMGENIX

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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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.