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HEMGENIX®-logo-bg HEMGENIX® (etranacogene dezaparvovec)

HELPFUL RESOURCES FOR YOUR PRACTICE

Find what you need to start your patients on HEMGENIX:

Optional AAV5 Neutralizing Antibody (NAb) Test

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Testing for AAV5 NAbs is not an eligibility requirement for HEMGENIX. If you choose to test your patient, please note that AAV5 NAbs do not exclude patients from receiving HEMGENIX.

Upon request, CSL Behring will make available a laboratory developed, CLIA-validated test that was used during the clinical trial. See below for details.

In the HOPE-B study, patients were assessed for AAV5 neutralizing antibodies using a clinical laboratory test, but patients were not excluded based on their test results nor are they excluded in the approved indication. However, one patient that did not respond to treatment was found to have an AAV5 neutralizing antibody titer level of 1:3212.

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AAV5 NAb TEST PROCESS

Step 1: Order a test kit

Step 1: Order a test kit

You can request the AAV5 Neutralizing Antibody (NAb) Test by calling 1-833-436-0021, Monday through Friday from 8 AM-8 PM ET.

You will receive the test kit(s) within 2-3 business days after placing your order.

The AAV5 Neutralizing Antibody (NAb) Test Kit contains:

  • 1 VACUETTE® SS Tube
  • 2 cryovials
  • 1 transfer pipette
  • 1 instruction for use
  • 2 specimen ID stickers
  • 1 biohazard bag
  • 1 Cryosleeve
  • 1 FedEx® air waybill
  • 1 return shipper (Styrofoam box and outer shipping container)

All kit components can be stored at 4°C-25°C until ready for collection. If additional time is necessary to prepare specimens for shipment, cryovials should remain in the freezer at -70°C or below, or placed into dry ice. Materials that may be required but are not provided: Needle, tube adapter, alcohol swab, bandage, gloves, gauze, tourniquet.

Do not use past the expiration date printed on label.

Step 2: Complete the AAV5 Test Requisition Form

Step 2: Complete the AAV5 Test Requisition Form

The AAV5 Test Requisition Form is conveniently located on a digital portal available to you.

  • Complete Part 1 of the Test Requisition Form on the partner lab’s digital portal
  • Once Part 1 is complete, you must print, sign, and give a hard copy of the Test Requisition Form to either your patient or a staff member to deliver directly to the lab collecting the sample
  • The lab will collect the sample (see Step 3 below) and then complete Part 2 of the Test Form
  • Once both Part 1 and Part 2 are complete, send both parts of the Test Form along with the sample to the partner lab
Questions?

Call HEMGENIX Connect℠ at 1-833-436-0021, Monday through Friday from 8 AM-8 PM ET.

Step 3: Collect test sample via blood draw

Step 3: Collect test sample via blood draw

Man standing with legs crossed
icon of sample tube
FILL SAMPLE TUBE
  • The VACUETTE® SS Tube and 2 cryovials in the kit are pre-labelled with a barcode that identifies the unique specimen
  • Using a solvent and water-resistant permanent marker, fill in the Precision for Medicine Patient ID (listed on Part 1 of the Test Requisition Form) and the date of collection (YYYY-MM-DD) where indicated on the labels
  • Record the date (YYYY-MM-DD) and time (24h clock, HH:MM) of collection, centrifugation and freezing on the Test Requisition Form. Make a copy of the completed Test Requisition Form for your records if required
  • Specimen collection and handling shall follow the instructions in the package insert for the Greiner Bio-One VACUETTE® Serum Tube
  • Once sample is collected, the manufacturer recommends inverting the tube 5-10 times
icon of clock
SET AND CENTRIFUGE
  • Allow blood to clot in upright position for a minimum of 30 minutes. Do not refrigerate prior to centrifugation. Centrifugation temperatures should be maintained at 15°C-25°C
  • Centrifuge sample—the recommended g-force or relative centrifugal force (rcf) is 1800 g and the recommended time of centrifugation is 10 minutes
icon of test tubes and dropper
TRANSFER TO CRYOVIALS
  • Using the transfer pipette, transfer serum from the SS Tube equally into the 2 cryovials. Discard SS Tube in an appropriate biohazard container or in accordance with local procedures
icon two hour countdown
FREEZE AND SHIP
  • The time from specimen collection to freezing should not exceed 2 hours
  • Store samples at -70°C or lower until ready for shipment
  • Prior to shipment of samples, make sure that there is sufficient dry ice available for packaging the shipment. It is recommended that a minimum of 5 kg of dry ice is used for routine shipments
  • Using the cryosleeve and biohazard bag provided, place the cryovials into the cryosleeve, and the cryosleeve into the biohazard bag. Seal the biohazard bag. Fill half of the cooler box with approximately 2.5 kg of dry ice
  • Place the completed Test Requisition Form in the biohazard bag using the document pouch, then place the biohazard bag into the return shipper. Fill up the rest of the cooler box with the remaining dry ice for a minimum total of 5 kg of dry ice, then close the return shipper and seal with packaging tape
  • Use the pre-printed FedEx Air Waybill found inside the return shipper for mailing. Take note of the FedEx tracking information for your records if required

Step 4: Review test results and discuss next steps with your patient

Step 4: Review test results and discuss next steps with your patient

In 2-3 weeks, your practice will receive an email letting you know a report providing the AAV5 Neutralizing Antibody (NAb) Test results for your patient is ready for your review on the digital portal

  • Test results are reported as AAV5 NAb titer, or no result. A reason will be provided if there is no result
  • In certain circumstances, there may be a need for retesting

Patient Eligibility Brochure

Patient Eligibility Brochure

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Information about patient eligibility and the AAV5 NAb test process is available in the Patient Eligibility Brochure.

If you have additional questions about the Test Requisition Form, test kits, or accessing the portal, please call HEMGENIX Connect℠ 1-833-436-0021, Monday through Friday from 8 AM-8 PM ET.

Man standing with legs crossed Download the brochure

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IMPORTANT SAFETY INFORMATION

Infusion Reactions

Perform baseline testing to select patients, including titer testing for Factor IX (FIX) inhibitor presence. Do not administer HEMGENIX®, etranacogene dezaparvovec-drlb, to patients with FIX inhibitors or a history of FIX inhibitors. Perform liver health assessments, consulting with a hepatologist if needed. Also perform laboratory tests to evaluate hepatitis B and C, and postpone treatment if patient has active infection, as this may reduce the efficacy of HEMGENIX and/or increase the risk of adverse reactions.

Warnings and Precautions

Hypersensitivity and Infusion Reactions

Infusion reactions, including hypersensitivity reactions and anaphylaxis, have occurred. Monitor during administration and for at least 3 hours after end of infusion. If symptoms occur, slow or interrupt administration. When symptoms have resolved, restart administration at a slower infusion rate.

Hepatotoxicity/Hepatocellular Carcinogenicity

Hepatotoxicity with elevated liver transaminase has occurred after HEMGENIX treatment. Monitor ALT levels once per week for 3 months and thereafter monthly up to 1 year after administration. Consider corticosteroid treatment should elevations occur and as clinically indicated.

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, consider liver ultrasound and alpha-fetoprotein testing following administration, and monitor for hepatocellular carcinomas for five years following administration of HEMGENIX.

Immune-Mediated Neutralization of the AAV5 vector capsid

Preexisting neutralizing anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with HEMGENIX, all patients developed neutralizing anti-AAV5 antibodies to AAV5 vector capsid.

Monitoring Laboratory Tests

Monitor patients regularly for FIX activity (eg, weekly for 3 months), especially when exogenous FIX is administered, as it may take several weeks following HEMGENIX administration before hemostatic control becomes apparent. Hemostatic support may be needed for some patients. Monitor patients through appropriate clinical observations and laboratory tests for the development of inhibitors to FIX.

Adverse Reactions

The most common adverse reactions (incidence ≥5% in clinical trials) are elevated ALT, headache, blood creatine kinase elevations, flu-like symptoms, infusion-related reactions, fatigue, nausea, malaise, and elevated AST.

Indication

HEMGENIX is 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.

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.