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HEMGENIX offers elevated and sustained factor IX
levels for years after a single infusion1

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Formulary kit

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

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Four-year data report

Four-year data now available

Take a closer look at HEMGENIX efficacy, safety, and durability at 4 years.1

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About hemophilia B

Hemophilia B is a rare, X-linked hereditary disorder characterized by bleeding, pain, and long-term complications.2-4 Hemophilia B is caused by a defect in a gene encoding coagulation factor IX, which is expressed primarily in hepatocytes. This mutation occurs in 5 per 100,000 male births.4-6 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.3,4,7

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?

At 4 years, a single dose of HEMGENIX was associated with:

Reduction In Factor IX Consumption

REDUCTION IN FACTOR
IX CONSUMPTION1

Mean factor IX activity icon

MEAN FACTOR
IX ACTIVITY1

Reduction in annualized bleed rate (ABR)

REDUCTION IN
ANNUALIZED BLEED
RATE (ABR)1

VS ROUTINE FACTOR
IX PROPHYLAXIS*

At 4 years, HEMGENIX continues to:

Hemgenix DNA image

Have NO reported serious treatment-related adverse reactions1

NOT be associated with the development of inhibitors to factor IX8

*Adjusted ABR for all bleeds decreased from an average of 4.16 for prophylaxis during the lead-in period to 1.63 in months 7 to 48 after treatment, a reduction of 61%. During months 7 to 18, ABR for all bleeds decreased by 64%.

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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The value of HEMGENIX

For a 1-million-member plan, it is estimated that 2 patients may qualify for HEMGENIX.† According to ICER’s analysis, HEMGENIX was projected to be a dominant treatment compared with 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 with the standard of care.9

ICER analysis projected factor IX levels

Abbreviation: 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. Data on file. Available from CSL Behring as DOF HGX-010 2. 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 3. Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020;105(3):545-553. doi:10.3324/haematol.2019.232132 4. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26(suppl 6):1-158. doi:10.1111/hae.14046 5. About hemophilia. Centers for Disease Control and Prevention. May 15, 2024. Accessed November 20, 2024. https://www.cdc.gov/hemophilia/about/ 6. 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 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; on behalf of the HOPE-B investigators. The phase 3 HOPE-B trial shows 4-year durability of sustained near-normal FIX activity, bleed protection and favourable safety in adults with severe or moderately severe haemophilia B. Presented at: 18th Annual Congress of the European Association for Haemophilia and Allied Disorders; February 4-7, 2025; Milan, Italy. 9. 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 November 20, 2024. 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.

©2025 CSL Behring LLC. The product information presented on this page is intended for US residents only.

USA-HGX-0934-MAR25

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