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Overview: Hemgenix is a one-time medicine used to treat certain people with hemophilia B. It is a gene therapy that helps your body make factor IX, which your body uses to make blood clots to stop bleeding. Common side effects may include headache, tiredness, and flu-like symptoms. Serious side effects are less common, but may include liver problems. Your health care provider will order certain tests to monitor your liver before and after receiving Hemgenix.

Uses

What is Hemgenix used for?

Hemgenix (etranacogene dezaparvovec-drlb) is commonly used for certain people who have a genetic blood disorder known as hemophilia B.

What to know about Hemgenix for hemophilia B.

How does Hemgenix work (mechanism of action)?

People with hemophilia B do not have enough of a particular clotting factor, known as factor IX. Your body uses factor IX to make blood clots.

Hemgenix is a type of gene therapy, which means that it adds a gene that helps you to make more factor IX. Once your treatment with Hemgenix is completed, the new gene is a permanent part of your body. Having enough factor IX may help to reduce the need for factor IX replacement transfusions in people with hemophilia B.

How is Hemgenix supplied (dosage forms)?

Hemgenix is available in an injectable form that is given by a health care provider.

How should I store Hemgenix?

Hemgenix is given by a health care provider in a hospital or clinic. You will not store it at home.

Side Effects

What are the most common side effects of Hemgenix?

The most common side effects of Hemgenix are listed below. Tell your health care provider if you have any of these side effects that bother you.

  • Liver problems (see below)
  • Headache
  • Flu-like symptoms
  • Infusion reactions (see below)
  • Tiredness
  • General feeling of being unwell, tired, or not having your usual energy

There may be other side effects of Hemgenix that are not listed here. Contact your health care provider if you think you are having a side effect of a medicine. In the U.S., you can report side effects to the FDA at www.fda.gov/medwatch or by calling 800-FDA-1088 (800-332-1088). In Canada, you can report side effects to Health Canada at www.health.gc.ca/medeffect or by calling 866-234-2345.

What are the serious side effects of Hemgenix?

While less common, the most serious side effects of Hemgenix are described below, along with what to do if they happen.

Severe Allergic Reactions. Hemgenix may cause allergic reactions, which can be serious. Get help right away if you have any of the following symptoms of a serious allergic reaction.

  • Breathing problems or wheezing
  • Racing heart
  • Fever or general ill feeling
  • Swollen lymph nodes
  • Swelling of the face, lips, mouth, tongue, or throat
  • Trouble swallowing or throat tightness
  • Itching or skin rash
  • Bumps on the skin called hives that can be red, pink, white, or brown depending on your skin tone
  • Nausea or vomiting
  • Dizziness, feeling lightheaded, or fainting
  • Stomach cramps
  • Joint pain

Liver Problems. Hemgenix may cause liver damage, also called hepatotoxicity. People with existing liver problems may have a higher risk. You will need to get frequent blood tests, called liver function tests (LFTs), to check the health of your liver before and after Hemgenix treatment. Keep all appointments, including those for lab work or other tests before and after receiving Hemgenix. Call your health care provider right away if you have any of the following symptoms of liver damage.

  • Nausea or vomiting
  • Stomach or belly pain
  • Fever
  • Weakness or unusual tiredness
  • Itching
  • Loss of appetite
  • Light-colored poop
  • Dark-colored urine (pee)
  • Your skin or the whites of your eyes turning yellowish in color (also called jaundice)

Infusion Reactions. Serious reactions can happen while you are receiving Hemgenix or within several hours after the infusion. Sometimes these can be serious. Your infusion may need to be slowed or stopped if you have a reaction during treatment. Your health care provider may need to give you medicines to help with the reaction if you have one. Tell your health care provider right away if you have any of the following symptoms during or after your infusion.

  • Chest tightness
  • Headache
  • Stomach pain
  • Feeling lightheaded
  • Fever, chills, or shivering
  • Flushing
  • Rash

Warnings & Precautions

Who should not use Hemgenix?

Allergies to Ingredients. People who are allergic to any of the following should not use Hemgenix.

  • Etranacogene dezaparvovec-drlb 
  • Hemgenix
  • Any of the ingredients in the specific product dispensed

Your pharmacist can tell you all of the ingredients in Hemgenix.

What should I know about Hemgenix before using it?

Hemgenix is usually given by a health care provider in a hospital or clinic. 

It can take a few weeks after receiving Hemgenix before your blood clotting improves. During that time it may be necessary for you to continue to receive Factor IX infusions for a few weeks after your dose of Hemgenix. Talk to your health care provider if you have concerns. 

Because of the way that Hemgenix delivers the gene therapy to your body, it may increase your risk for liver cancer, especially if you have other risk factors. Before using Hemgenix, talk to your health care provider about these risks. Keep all appointments for lab tests and any other monitoring before and after receiving Hemgenix.

After receiving Hemgenix, you should not donate blood, organs, tissues, or other cells. Talk to your health care provider if you have concerns. 

What should I tell my health care provider before using Hemgenix?

Tell your health care provider about all of your health conditions and any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. This will help them know if Hemgenix is right for you.

In particular, make sure that you discuss any of the following.

Current and Past Health Conditions. Tell your health care provider if you have ever received Hemgenix or any of the following.

  • Known factor IX inhibitors, which are antibodies made by the immune system that attack replacement factor IX and can affect how well treatments work 
  • Liver cancer or other liver problems, such as increased liver enzymes or hepatitis

Pregnancy. People who can become pregnant are not usually prescribed Hemgenix. 

Breastfeeding. People who can breastfeed are not usually prescribed Hemgenix. 

How can I get more information about Hemgenix?

For more information about Hemgenix, you can visit the drugmaker’s website at www.hemgenix.com or call them at 888-508-6978 or 833‑436‑0021.

Interactions

Does Hemgenix interact with foods or drinks?

There are no known interactions between Hemgenix and foods or drinks.

It is unknown if drinking alcohol will affect Hemgenix, but both Hemgenix and alcohol can lead to liver problems. It is best to limit the amount of alcohol you drink.

Does Hemgenix interact with other medicines (drug interactions)?

There are no known interactions between Hemgenix and other medicines. Always tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using.

Does interact with other drugs you are taking?

Enter your medication into the WebMD interaction checker

Overdose/Missed Dose

What should I do if I accidentally use too much Hemgenix?

If you or someone else has used too much Hemgenix, get medical help right away, call 911, or contact a Poison Control center at 800-222-1222.

What should I do if I miss a dose of Hemgenix?

Hemgenix is a one-time treatment. It is usually given by a health care provider in a hospital or clinic. If you miss the appointment to get your Hemgenix dose, contact your health care provider to reschedule.