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In these short videos, the experts explore where and why gene therapy fits into the hemophilia B treatment landscape, while discussing the clinical evidence and practical considerations supporting the use of HEMGENIX. Watch them in order or select topics of interest.
This is a paid promotional program presented by the speakers on behalf of CSL Behring LLC.
Exploring the Unmet Needs and Disease Burden in Hemophilia B Treatment
Review the evolution of treatments for hemophilia B and find out where opportunity remains in meeting the needs of patients.
Why Is Hemophilia B a Good Target for Gene Therapy?
Understand the technology and clinical value of AAV vectors used to deliver gene transfer therapy.
Clinical Evidence Supporting the Use of HEMGENIX for Appropriate Patients
Hear the experts discuss the efficacy and safety results from the phase 2 and 3 (HOPE-B) trials that led to FDA approval of HEMGENIX.
Which Patients Are Eligible for HEMGENIX?
Discover the key characteristics for patient selection and additional clinical factors to consider when identifying patients who may be candidates for HEMGENIX.
Considerations for Practice Readiness
Learn about the recommended steps in prescribing HEMGENIX and the support services available through the entire HEMGENIX journey.
Expert Insights: Addressing the Unmet Need and Impacting the Lives of Patients
Listen as the experts discuss the clinical value of gene therapy and how HEMGENIX can impact the lives of patients with hemophilia B.
Guy Young, MD
Professor of Pediatrics, University of Southern California; Director, Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles
Chief, Pediatric Hematology; Director, Hemophilia Treatment Center, Mississippi Center for Advanced Medicine
Vice President, Medical Affairs, North America, CSL Behring
Warning and Precautions
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.
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.
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.
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:
HEMGENIX is for single use intravenous infusion only.
Please see full prescribing information for HEMGENIX.