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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

*Elevated factor IX levels have been observed annually.

§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.

*Elevated factor IX levels have been observed annually.

§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 annualized 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 versus HEMGENIX

A one-time infusion of HEMGENIX also showed:

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

†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).

94%

Of patients discontinued

Factor IX prophylaxis

and remained prophylaxis-free1‡

With Hemgenix,

Most patients eliminated routine factor IX prophylaxis infusions1

Pie chart filled 94%
Man standing with hands clasped

Patient portrayal

Discontinued Routine Factor IX Prophylaxis

AND REMAINED PROPHYLAXIS-FREE1

‡ 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).

*Elevated factor IX levels have been observed annually.

†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).

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 were monitored for up to 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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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. HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients. CTG Labs - NCBI. June 26, 2018. Accessed May 18, 2023. https://clinicaltrials.gov/study/NCT03569891.

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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.