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First gene therapy for hemophilia B

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Formulary kit

Review product-specific resources for the first gene therapy for hemophilia B.

Product Fact Sheet
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Prescribing Information
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P&T Summary
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Product Information
for Hospitals (AHFS)
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Billing & Coding Guide
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FormularyDecisions® members can download the HEMGENIX Dossier and additional HEMGENIX resources from FormularyDecisions.com.

Explore value perspectives

Explore value perspectives

Get payer and manufacturer perspectives on how to evaluate breakthrough treatments like HEMGENIX in the report The Value of Innovative Medicine.

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About hemophilia B

Hemophilia B is a rare, X-linked hereditary disorder characterized by bleeding, pain, and long-term complications.1-3 Hemophilia B is caused by a defect in a gene encoding coagulation factor IX, which is expressed primarily in hepatocytes and occurs in 5 per 100,000 male births.3-6 The severity of hemophilia B typically correlates to native coagulation factor activity. Severe hemophilia B is characterized by spontaneous and/or traumatic bleeding into joints, muscles, and internal organs, and can result in reduced life expectancy.2,3,7

What is HEMGENIX?

HEMGENIX 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.

Please see full prescribing information for HEMGENIX.
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What are the key benefits?

HEMGENIX is the first gene therapy approved to treat hemophilia B and is available in the United States.

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MEAN FACTOR IX ACTIVITY

sustained at 3 years8

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REDUCTION IN FACTOR IX CONSUMPTION8

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SIGNIFICANT REDUCTION IN ANNUAL BLEED RATE (ABR)

vs routine factor IX prophylaxis8*

*Annual bleed rate (ABR) for all bleeds decreased from an average of 4.17 for prophylaxis during the lead-in period to 1.52 in months 7 to 36 after treatment, sustaining the same bleed rate reduction that satisfied the primary endpoint during months 7 to 18.

Three-year data report

Three-year data report

Take a closer look at HEMGENIX’s efficacy, safety, and durability at 3 years.

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The value of HEMGENIX

For a one-million-member plan, it is estimated that 2 patients may qualify for HEMGENIX.9† According to ICER’s analysis, HEMGENIX was projected to be a dominant treatment compared to factor IX prophylaxis with lower costs and higher quality-adjusted life years. ICER’s base-case analysis projected HEMGENIX use would result in >$6 million in savings over the lifetime of a hemophilia B patient compared to standard of care.10

ICER analysis projected factor IX levels

Abbreviations: FIX, factor IX; ICER, Institute for Clinical and Economic Review.


†Approved indication may also include people who are not currently on prophylaxis. See approved prescribing information for appropriate patient selection.

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Important Safety Information

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.

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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.

References: 1. Liras A, Segovia C, Gabán AS. Advanced therapies for the treatment of hemophilia: future perspectives. Orphanet J Rare Dis. 2012;7:97. doi:10.1186/1750-1172-7-97 2. Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020;105(3):545-553. doi:10.3324/haematol.2019.232132 3. Srivastava A, Santagostino E, Dougall A, et al; WFH Guidelines for the Management of Hemophilia panelists and co-authors. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26(suppl 6):1-158. doi:10.1111/hae.14046 4. What is hemophilia? Centers for Disease Control and Prevention. Updated July 17, 2020. Accessed March 7, 2022. https://www.cdc.gov/ncbddd/hemophilia/facts.html 5. Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540-546. doi:10.7326/M19-1208 6. A new study of hemophilia occurrence finds many more cases in the United States. Centers for Disease Control and Prevention. Reviewed August 8, 2022. Accessed January 18, 2023. https://www.cdc.gov/ncbddd/hemophilia/features/keyfinding-hemophilia-occurrence-US.html 7. Learn more about hemophilia B. The Coalition for Hemophilia B. Accessed March 7, 2022. https://www.hemob.org/about-hemophilia-b 8. Pipe SW, van der Valk P, Verhamme P, et al. Long-term bleeding protection, sustained FIX activity, reduction of FIX consumption and safety of hemophilia B gene therapy: results from the HOPE-B trial 3 years after administration of a single dose of etranacogene dezaparvovec in adult patients with severe or moderately severe hemophilia B. Abstract presented at: 65th American Society of Hematology Annual Meeting; December 9-12, 2023; San Diego, CA. 9. Data on file. Available from CSL Behring as DOF ETZ-001. 10. Tice JA, Walton S, Herce-Hagiwara B, et al; Institute for Clinical and Economic Review. Gene Therapy for Hemophilia B and An Update on Gene Therapy for Hemophilia A: Effectiveness and Value. Final evidence report. December 22, 2022. Accessed March 6, 2023. https://icer.org/wp-content/uploads/2022/12/ICER-Hemophilia-Policy-Recommendations-122222.pdf

HEMGENIX is manufactured by uniQure Inc. and distributed by CSL Behring LLC.

HEMGENIX® is a registered trademark of CSL Behring LLC.

All other trademarks are property of their respective owners.

©2024 CSL Behring LLC. The product information presented on this page is intended for US residents only.

USA-HGX-0958-MAR24

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