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Overview: Zynteglo is a gene therapy that is used to treat certain people with a blood disorder called beta-thalassemia. It changes your stem cells to provide a gene that helps to form functional hemoglobin. In order to receive Zynteglo, you will need to undergo multiple different procedures and treatments, including a bone marrow transplant. Altogether, this treatment may take more than six months to complete. Each of these procedures and treatments carries its own risks and side effects. Make sure that you talk with your health care provider to understand the entire process.

Uses

What is Zynteglo used for?

Zynteglo (betibeglogene autotemcel) is commonly used to treat certain people with a blood disorder known as beta-thalassemia.

Zynteglo may also be used for other conditions as determined by your health care provider.

How does Zynteglo work (mechanism of action)?

People with beta-thalassemia have changes (mutations) in a gene in their red blood cells. This changes the way the hemoglobin in their red blood cells forms and functions, which can cause many serious health problems. Hemoglobin is the substance in your red blood cells that carries oxygen. 

Zynteglo is a type of gene therapy, which means that it adds a gene that helps you to form hemoglobin that functions normally. Once your treatment with Zynteglo is completed, the new gene is a permanent part of your body. Having functional hemoglobin may help to reduce the need for blood transfusions in people with beta-thalassemia.

How is Zynteglo supplied (dosage forms)?

Brands and other names

  • Zynteglo

Dosage forms and strengths

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

How should I store Zynteglo?

Zynteglo 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 Zynteglo?

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

  • Mouth or lip sores
  • Severely low platelet levels (see below)
  • Anemia
  • Low white blood cell levels (see below)
  • Nausea or vomiting
  • Fever
  • Hair loss
  • Nosebleeds
  • Stomach or muscle pain
  • Cough
  • Headache

There may be other side effects of Zynteglo 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. 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 Zynteglo?

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

Severe Allergic Reactions. Zynteglo 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
  • 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

Low White Blood Cell Levels. After treatment with Zynteglo, you may have a low level of white blood cells for a period of time, including a type of white blood cell (called neutrophils), which is known as neutropenia. White blood cells help prevent infection. Having a low level may increase your risk of infection. Tell your health care provider right away if you have any of the following symptoms of low white blood cell levels.

  • Infection
  • Tiredness
  • Sore throat, cough, or shortness of breath
  • Mouth or throat sores
  • Swollen lymph nodes
  • Fever, chills, severe headache, or sweats

Severely Low Platelet Levels. After treatment with Zynteglo, you may have a severely low number of platelets in your blood, also called thrombocytopenia, for a period of time. Platelets help form blood clots. Having a low level of platelets may increase your risk of bleeding. Tell your health care provider right away or get emergency help if you have any of the following symptoms of low platelet levels.

  • Bleeding that does not stop
  • Nosebleeds
  • Easy bruising or bleeding
  • Blood in your stool (poop), pee, or vomit
  • Coughing up blood
  • Severe headache
  • Unusual stomach or back pain

Cancer. Zynteglo may increase the risk for certain types of blood cancers. Your health care provider will monitor you for signs of blood cancer for at least 15 years after you receive Zynteglo. Keep all appointments for blood work and other checkups during this time. Tell your health care provider about any new symptoms that you may get. 

Warnings & Precautions

Who should not use Zynteglo?

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

  • Betibeglogene autotemcel
  • Dimethylsulfoxide (DMSO)
  • Zynteglo
  • Any of the ingredients in the specific product dispensed

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

What should I know about Zynteglo before using it?

Zynteglo is a personalized gene therapy that is given by a health care provider in a hospital. Make sure that you discuss the entire treatment plan with your health care provider before it starts.

Before your treatment with Zynteglo, your stem cells will be collected from your body and sent to a lab. It may take up to three months for your edited stem cells to be prepared in the lab. Before your stem cells are collected, you may need to stop taking certain medicines. You may also need to receive a blood transfusion. It is important to follow all instructions and keep all appointments with your health care provider during this process.

Before receiving treatment with Zynteglo, your health care provider will give you “conditioning” medicine that will clear all of the bone marrow cells from your body. After this is done, you will be at high risk for infection and bleeding and will need to stay in the hospital for the rest of your treatment. While you are there, you may need to take medicines to prevent or treat side effects from the conditioning medicine. 

Once your bone marrow is cleared, your edited stem cells (Zynteglo) will be infused into your vein (this is known as a bone marrow transplant). These stem cells will then rebuild your bone marrow, a process called engraftment. It can take four to six weeks for this to happen. You may need to remain in the hospital for this entire period. While you are waiting for engraftment, you will have very little or no white blood cells or platelets, which means you will be at high risk for infection and bleeding.

In rare cases, your edited stem cells may not engraft at all. This is called engraftment failure. If this happens, your health care provider will infuse your original stem cells back into your body. This means that your original stem cells will replace your edited stem cells, and you will not get any benefit from Zynteglo.

The “conditioning” medicine that is used to clear your bone marrow cells from your body may make it difficult for you to get pregnant or get your partner pregnant. If you want to have children in the future, speak with your health care provider about your options.

If you are able to get pregnant or get someone else pregnant, you should use an effective form of birth control throughout your entire treatment process with Zynteglo. This means that you should start birth control when you are getting ready to have your stem cells collected and continue using birth control for at least six months after receiving Zynteglo.

Do not donate any blood, tissues, cells, or organs to another person after you get Zynteglo. 

Zynteglo will not give you HIV but may cause you to test positive on a blood test for HIV when a certain type of test is used (a PCR-based test). If you need to be tested for HIV, speak with your health care provider to make sure an appropriate test is used.

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

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 determine if Zynteglo is right for you.

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

  • Current infection, infection that keeps coming back, or low levels of white blood cells
  • Bleeding problems or low levels of platelets
  • HIV or AIDS
  • Prior bone marrow transplant (BMT), also called a hematopoietic stem cell transplant (HSCT)

Vaccines. Talk with your health care provider about any vaccines that you may need. You should not receive certain vaccines right before or while using Zynteglo. Tell your health care provider about any vaccines you have recently received or have scheduled.

Other Medicines and Supplements. Zynteglo may interact with other medicines and supplements. Before receiving Zynteglo, tell your health care provider about any prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, and other supplements you are using. See the Interactions section for more details.

Pregnancy. Do not get Zynteglo if you are pregnant or trying to get pregnant. It is not known if or how Zynteglo could affect pregnancy or harm a fetus. If you are able to get pregnant, your health care provider will confirm that you are not pregnant before you begin treatments related to Zynteglo. You should use an effective form of  birth control from the time that your stem cells are collected until six months after you have received your treatment with Zynteglo. 

Breastfeeding. It is not known if Zynteglo passes into breast milk. Tell your health care provider if you are breastfeeding or plan to breastfeed. Your health care provider will let you know if you should get Zynteglo while breastfeeding or breastfeed after getting this medicine.

How can I get more information about Zynteglo?

For more information about Zynteglo, you can visit the drugmaker’s website at www.zynteglo.com or call them at 833-666-2583.

Interactions

Does Zynteglo interact with foods or drinks?

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

It is unknown if drinking alcohol will affect Zynteglo, but drinking can slow your immune response and make it harder for your body to fight infection. Follow your health care provider’s guidance about limiting or avoiding alcohol while you are receiving treatments related to Zynteglo. 

Does Zynteglo interact with other medicines (drug interactions)?

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.

In particular, make sure that you discuss if you are using any of the following before beginning treatments related to Zynteglo.

  • A medicine to prevent HIV
  • Deferasirox (Exjade, Jadenu), deferiprone (Ferriprox), or deferoxamine (Desferal), which are medicines used to lower the amount of iron in your body
  • Hydroxyurea (Hydrea, Siklos, Xromi), which is a medicine used to treat sickle cell disease and some cancers
  • Filgrastim (Neupogen) or sargramostim (Leukine), which are medicines that can be used to help your bone marrow make more cells

This may not be a complete list of medicines that can interact with Zynteglo. Always check with your health care provider.

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 Zynteglo?

If you or someone else has used too much Zynteglo, 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 Zynteglo?

Zynteglo and its related treatments are administered in a health care facility by a health care provider. Keep all appointments to help your treatment work. If you need to miss an appointment, contact your health care provider right away.