In clinical studies, HEMGENIX was safe and effective, with no treatment-related serious adverse events and no development of factor IX inhibitors reported.
Alanine aminotransferase increased
Blood creatine kinase increased
Infusion-related reactions† (see below)
Aspartate aminotransferase increased
Infusion-related reactions (IRRs) occurred in 19/57 (33%) of patients. The majority (58%) of patients recovered on the day of or one day after infusion.
In the event of an IRR during administration, the infusion may be slowed or temporarily stopped and restarted at a slower rate once the infusion reaction is resolved. Treatment with a corticosteroid or antihistamine may be considered for management of IRR.
In the clinical studies, ALT elevation occurred in 24/57 (42%) of patients.
See the steps to administering HEMGENIXLearn about dosing
Which of your patients may benefit from HEMGENIX?Patient eligibility
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.