Betibeglogene autotemcel Injection

Betibeglogene autotemcel injection is used to treat anemia (a lower than normal number of red blood cells) in people who require regular blood transfusions to treat beta thalassemia (Cooley's anemia; an inherited condition that causes a low number of red blood cells). Betibeglogene autotemcel injection is in a class of medications called autologous gene therapy, a type of medication prepared using cells from the patient's own blood stem cells. It works by helping your body to produce enough red blood cells so that regular blood transfusions are not necessary.

Betibeglogene autotemcel injection comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a hospital. Betibeglogene autotemcel injection is given as a one-time dose administered over up to 2 hours. Before you receive your betibeglogene autotemcel dose, your doctor or nurse will administer other chemotherapy medications to prepare your body for betibeglogene autotemcel.

Before your dose of betibeglogene autotemcel injection is to be given, a sample of your blood stem cells will be taken at a hospital or treatment center using a process called mobilization and apheresis (a process that removes blood stem cells from the body). This process usually takes about 1 week and may need to be repeated. The sample will be sent to the manufacturer to prepare a dose of betibeglogene autotemcel injection. It will take about 70–90 days from the time that your blood stem cells are collected before they are ready to be given to you. Because this medication is made from your own cells, it must be given only to you.

You will have to stay in the hospital for about 3–6 weeks after you receive your betibeglogene autotemcel dose to monitor you for any side effects. Talk to your doctor about how to prepare for mobilization and apheresis and what to expect during and after the procedure.

Betibeglogene autotemcel injection may cause infusion reactions, which may occur within 24 hours of receiving your dose. Tell your doctor or nurse if you experience any of the following symptoms: fast or irregular heartbeat or stomach pain.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before recieving betibeglogene autotemcel injection,

• tell your doctor and pharmacist if you are allergic to betibeglogene autotemcel, dimethyl sulfoxide (DMSO), any other medications, or any of the ingredients in betibeglogene autotemcel injection. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• tell your doctor if you have or have ever had seizures, hepatitis B or C (a virus that infects the liver and may cause severe liver damage), human immunodeficiency virus (HIV), or kidney or liver disease.
• tell your doctor if you are pregnant or plan to become pregnant. You should not become pregnant while you are receiving betibeglogene autotemcel injection. You will need to have a pregnancy test before you start treatment, and you should use birth control to prevent pregnancy during your treatment and for 6 months after you receive betibeglogene autotemcel injection. If you are a male with a female partner who may become pregnant, you should use birth control to prevent pregnancy during you treatment and for 6 months after you receive betibeglogene autotemcel injection. Talk to your doctor about birth control methods that will work for you. If you or your partner become pregnant while receiving betibeglogene autotemcel injection, call your doctor immediately. Betibeglogene autotemcel injection may cause fetal harm.
• tell your doctor if you are breastfeeding or plan to breastfeed. You should not breastfeed while your are receiving betibeglogene autotemcel injection therapy.
• do not donate blood, organs, tissues, or cells for transplantation after you receive betibeglogene autotemcel injection.

Unless your doctor tells you otherwise, continue your normal diet.

Betibeglogene autotemcel injection may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• leg or arm pain
• sores in the mouth and throat
• hair loss
• diarrhea
• nausea
• vomiting
• constipation
• heartburn
• decreased appetite
• excessive tiredness
• changes in skin color

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately or get emergency medical treatment:

• unusual bruising or bleeding, nose bleeding, severe headache, blood in urine, stool, or vomit, coughing up blood, or unusual abominal or back pain
• fever, sore throat, ongoing cough and congestion, or other signs of infection
• hives; rash; itching; swelling of the lips, tongue, or throat; shortness of breath or difficulty breathing

Betibeglogene autotemcel injection may increase the risk that you will develop blood cancers. Your blood will be monitored at least yearly for at least 15 years after your treatment with betibeglogene autotemcel injection. Talk to your doctor about the risks of taking this medication.

Betibeglogene autotemcel injection may cause other side effects. Call your doctor if you have any unusual problems while receiving this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Keep all appointments with your doctor and the laboratory. Your doctor will may order certain lab tests to check your body's response to betibeglogene autotemcel injection.

Before having any laboratory test, tell your doctor and the laboratory personnel that you are receiving betibeglogene autotemcel injection. You should know that you may have a false-positive test for HIV after you receive betibeglogene autotemcel injection.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

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