sustained at 2 years
† The full analysis set included 54 patients dosed. Uncontaminated factor IX activity values exclude measurements within five half-lives of factor IX replacement therapy. Contaminated and missing values are not shown here. Specifically, the number of subjects excluded for contamination with factor IX replacement therapy at month 6, month 12, month 18, and month 24, were 3, 3, 3, 2, respectively.
vs. routine factor IX prophylaxis*
Reduction in ABR (N=54)
A one-time infusion of HEMGENIX also showed:
and remained prophylaxis-free†
AND REMAINED PROPHYLAXIS-FREE
To demonstrate non-inferiority of annualized bleeding rate (ABR) during months 7–18 after HEMGENIX treatment compared with ABR during the lead-in period.
Preexisting anti-AAV5 NAbs were assessed but not used as an exclusion criterion, and no prophylactic immunosuppression was required2
See the safety profile of HEMGENIXExplore safety data
Learn how to store, prep, and infuse HEMGENIXAdministration instructions
Reference: 1. Miesbach W, Frank WG, Leebeek FWG, Recht M, et al; for the HOPE-B Investigators. Final analysis from the pivotal phase 3 HOPE-B gene therapy trial: stable steady-state efficacy and safety of etranacogene dezaparvovec in adults with severe or moderately severe haemophilia B. Presented at: 15th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2022); February 2-4, 2022; Virtual.
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.