Thursday, May 29, 2025 at 2-3pm ET                             Pricing: Free for Members and Non-Members

Please note that this session is not accredited for continuing pharmacy education credit.

Program Description

CSL Behring is proud to present a webinar highlighting the 4-year milestones for HEMGENIX^® (etranacogene dezaparvovec-drlb), a one-time gene therapy approved for hemophilia B. This virtual presentation will explore the clinical and economic value of HEMGENIX^® vs the current standard of care — Q&A to follow

This webinar is sponsored, developed, and presented by the sponsor. The content of this Science & Innovation Webinar and opinions expressed by the presenters are those of the sponsor or presenters and not of AMCP.

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.

Sponsored by:

For individuals from the media/press interested in attending, please email media@amcp.org.