Gene Therapy Research in Hemophilia
World Federation of Hemophilia (WFH) Gene Therapy for Hemophilia
For over 50 years, the World Federation of Hemophilia (WFH) has worked globally to achieve treatment for all people with hemophilia and other inherited bleeding disorders, no matter where they live in the world.
International Society on Thrombosis and Haemostasis (ISTH): Gene Therapy in Hemophilia
The International Society on Thrombosis and Haemostasis (ISTH) advances the understanding, prevention, diagnosis, and treatment of conditions related to thrombosis and hemostasis.
American Thrombosis & Hemostasis Network
The American Thrombosis and Hemostasis Network (ATHN) is a nonprofit organization dedicated to improving the lives of people affected by bleeding and clotting disorders.
Foundation for Women & Girls With Bleeding Disorders
The Foundation for Women & Girls With Blood Disorders works to ensure all women and girls with blood disorders are correctly diagnosed and optimally managed at every life stage.
National Hemophilia Foundation
The National Hemophilia Foundation (NHF) is dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy, and research.
American Society of Hematology
With more than 18,000 members from nearly 100 countries, the ASH is the world's largest professional society serving both clinicians and scientists around the world who are working to conquer blood diseases.
Hemophilia Federation of America
Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
The Anticoagulation Forum is a nonprofit organization of multidisciplinary health care professionals improving the quality of care for patients taking antithrombotic medications.
American Association of Blood Banks
The AABB is an international, not-for-profit Association representing individuals and institutions involved in the fields of transfusion medicine and biotherapies.
Important Safety Information for HEMGENIXWarning 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.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 for HEMGENIX
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.