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hemgenix-logo-bg mobile-menu-bg HEMGENIX® (etranacogene dezaparvovec-drlb)

GROUNDBREAKING SCIENCE THAT CAN ENABLE YOU TO CREATE YOUR OWN FACTOR IX

DNA spiral with people hiking

Patient portrayal; HEMGENIX not intended for women

HEMGENIX is the first ever gene therapy for hemophilia B

HEMGENIX uses a gene therapy approach called gene transfer. This approach aims to introduce a working, or functional, gene into the body to produce functional factor IX and support natural blood-clotting ability.

Learn how HEMGENIX can enable people to make their own factor IX

HOW HEMGENIX WORKS

Step 1
gene therapy equation icons

The working gene is delivered to the liver by inserting it into an inactive viral shell, known as the vector. HEMGENIX uses an adeno-associated viral vector serotype 5, also called an AAV5 vector.

Step 2
IV infusion bag icon

Administered as an IV infusion, HEMGENIX heads toward the liver with the working gene.

Step 3
Liver generating FIX

Once it arrives in the liver, the working gene’s instructions are unloaded so that you can produce elevated and protective factor IX (FIX) on your own.

HEMGENIX is the first ever FDA-approved gene therapy for hemophilia B

See study results

Find out what you can expect from treatment

Watch the video
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IMPORTANT SAFETY INFORMATION

What is HEMGENIX?

HEMGENIX®, etranacogene dezaparvovec-drlb, is a one-time gene therapy for the treatment of adults with hemophilia B who:

  • Currently use Factor IX prophylaxis therapy, or
  • Have current or historical life-threatening bleeding, or
  • Have repeated, serious spontaneous bleeding episodes.

HEMGENIX is administered as a single intravenous infusion and can be administered only once.

What medical testing can I expect to be given before and after administration of HEMGENIX?

To determine your eligibility to receive HEMGENIX, you will be tested for Factor IX inhibitors. If this test result is positive, a retest will be performed 2 weeks later. If both tests are positive for Factor IX inhibitors, your doctor will not administer HEMGENIX to you. If, after administration of HEMGENIX, increased Factor IX activity is not achieved, or bleeding is not controlled, a post-dose test for Factor IX inhibitors will be performed.


HEMGENIX may lead to elevations of liver enzymes in the blood; therefore, ultrasound and other testing will be performed to check on liver health before HEMGENIX can be administered. Following administration of HEMGENIX, your doctor will monitor your liver enzyme levels weekly for at least 3 months. If you have preexisting risk factors for liver cancer, regular liver health testing will continue for 5 years post-administration. Treatment for elevated liver enzymes could include corticosteroids.

What were the most common side effects of HEMGENIX in clinical trials?

In clinical trials for HEMGENIX, the most common side effects reported in more than 5% of patients were liver enzyme elevations, headache, elevated levels of a certain blood enzyme, flu-like symptoms, infusion-related reactions, fatigue, nausea, and feeling unwell. These are not the only side effects possible. Tell your healthcare provider about any side effect you may experience.

What should I watch for during infusion with HEMGENIX?

Your doctor will monitor you for infusion-related reactions during administration of HEMGENIX, as well as for at least 3 hours after the infusion is complete. Symptoms may include chest tightness, headaches, abdominal pain, lightheadedness, flu-like symptoms, shivering, flushing, rash, and elevated blood pressure. If an infusion-related reaction occurs, the doctor may slow or stop the HEMGENIX infusion, resuming at a lower infusion rate once symptoms resolve.

What should I avoid after receiving HEMGENIX?

Small amounts of HEMGENIX may be present in your blood, semen, and other excreted/secreted materials, and it is not known how long this continues. You should not donate blood, organs, tissues, or cells for transplantation after receiving HEMGENIX.

Please see full prescribing information for HEMGENIX.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

You can also report side effects to CSL Behring’s Pharmacovigilance Department at 1-866-915-6958.