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HEMGENIX 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.
What is HEMGENIX?

HEMGENIX is an in vivo gene therapy that introduces a functional copy of the F9 gene, and after single intravenous infusion results in cell transduction and increase in circulating factor IX activity in patients with hemophilia B.

HEMGENIX consists of a liver-directed adeno-associated viral vector serotype 5 (AAV5) containing the factor IX-Padua gene cassette.

  • The AAV5 is nonreplicating and has a serotype-specific tropism for hepatocytes, where factor IX is naturally produced.
  • The highly active factor IX-Padua variant has been shown to generate 5-8 times higher mean endogenous factor IX activity than the wild-type gene. The factor IX-Padua gene is under the control of a liver-specific promoter.

With HEMGENIX, the factor IX-Padua gene is contained within the AAV5 vector to introduce a functional copy of the F9 gene into a patient’s hepatocytes.

AAV=adeno-associated virus; FIX=clotting factor IX.
1. D’Avola D, et al. J Hepatol. 2016;65:777–783; 2. Miesbach W, et al. Blood. 2018;131:1022–1031; 3. Pipe SW, et al. Poster presented at 61st ASH meeting, 2019.

HEMGENIX is provided as a customized kit containing 10-48 vials of HEMGENIX, corresponding to the dosing requirement for the individual patient, depending on their body weight. HEMGENIX vials contain no less than 10-mL of extractable volume and are for single use only.

HEMGENIX Example Dose Calculation

1. The recommended dose of HEMGENIX is 2 x 1013 genome copies per kg of body weight (or 2 mL/kg of body weight) administered as an IV infusion after dilution with 0.9% sodium chloride solution (normal saline)

Calculate the dose as follows:
       Dose (in mL) = patient body weight (in kg) x 2

For example, for a patient with a body weight of 85 kg, the dose would be:
       85 x 2 = 170 mL

A patient with a body weight of 128 kg would require:
       128 x 2 = 256 mL

2. Number of HEMGENIX vials needed = HEMGENIX dose (in mL) divided by 10 (round up to the next whole number of vials).  The total volume of the patient's HEMGENIX dose to be diluted may be less than the total volume of vials needed.

Patient Weight HEMGENIX dose (mL) (body weight multiplied by 2) Number of vials needed [HEMGENIX dose (mL) divided by 10, then rounded up]
85 kg 170 mL 17
128 kg 256 mL 26

 

3. Prior to dilution, withdraw the volume of the calculated dose from the 500 mL infusion bag(s) of 0.9% normal saline solution, depending on patient’s body weight

- For patients with a body weight of <120 kg, withdraw saline  volume equal to the total HEMGENIX dose in one 500 mL 0.9% normal saline infusion bag
For patients with a body weight of ≥120 kg, withdraw half of the dose equivalent volume from each of the two 500 mL 0.9% normal saline infusion bags

4. The volume of the required dose is then added to the infusion bag(s) to bring the total volume in each infusion bag back to 500 mL

85 kg Patient 128 kg Patient

Recommended dose = 85 kg x 2 mL/kg = 170 mL

Dilute the 170 mL of HEMGENIX in one 500 mL 0.9% normal saline solution infusion bag

Recommended dose = 128 kg x 2 mL/kg = 256 mL

Dilute the 256 mL of HEMGENIX in two 500 mL
0.9% normal saline solution infusion bags
(128 mL in each bag)

Handling 

Storage Image 1
Provided in a sealed biohazard bag as a customized kit depending on the patient’s body weight, each kit contains 10 to 48 vials (10 mL each)
Storage Image 2
Cold chain storage and handling required. Product is shipped at 2°C to 8°C (36°F to 46°F)
Storage Image 3
Store HEMGENIX in a refrigerator at 2°C to 8°C (36°F to 46°F) in the original carton within the biohazard bag. Protect from light until time of dilution and administration. Do not freeze.
Storage Image 4
After dilution, store in the infusion bag protected from light for up to 24 hours at 15°C to 25°C (59°F to 77°F) after the dose preparation

Please see Important Safety Information for HEMGENIX below

Important Safety Information for HEMGENIX

Warning and Precautions

Infusion Reactions

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.

Hepatotoxicity/Hepatocellular Carcinoma

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.

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For U.S. Healthcare Professionals only

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