Pronunciation:

be-ti-BEG-loe-jeen aw-toe-TEM-sel

Brand Names:

• Zynteglo

Dosage Forms:

• Suspension

Uses of This Medicine:

Betibeglogene autotemcel injection is used to treat beta thalassemia in patients who need regular red blood cell (RBC) transfusions.

This medicine is to be given only by or under the immediate supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of betibeglogene autotemcel injection in children younger than 4 years of age. Safety and efficacy have not been established.

Older adults—

Appropriate studies on the relationship of age to the effects of betibeglogene autotemcel injection have not been performed in the geriatric population.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are receiving this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Adenovirus Vaccine, Live
• Bacillus of Calmette and Guerin Vaccine, Live
• Cholera Vaccine, Live
• Dengue Tetravalent Vaccine, Live
• Ebola Zaire Vaccine, Live
• Influenza Virus Vaccine, Live
• Measles Virus Vaccine, Live
• Mumps Virus Vaccine, Live
• Poliovirus Vaccine, Live
• Rotavirus Vaccine, Live
• Rubella Virus Vaccine, Live
• Smallpox Monkeypox Vaccine, Live Non-Replicating
• Smallpox Vaccine
• Typhoid Vaccine, Live
• Varicella Virus Vaccine, Live
• Yellow Fever Vaccine
• Zoster Vaccine, Live

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Kidney disease or
• Liver disease—Use with caution. Your doctor will check if you have these conditions before receiving this medicine.

Proper Use of This Medicine:

A doctor or other trained health professional will give you this medicine in a hospital. It is given through a needle placed in a vein. This medicine must be given slowly, so your IV tube will need to stay in place for at least 4 hours.

This medicine comes with patient information leaflet. Read and follow these instructions carefully. Ask your doctor or pharmacist if you have any questions.

You may also receive other medicines (eg, cancer medicine) before receiving this medicine.

This medicine is made specifically for each patient, using the patient's own blood cells. Your doctor will collect your blood through a procedure called mobilization and apheresis. Talk with your doctor if you have any concerns about this.

Your doctor will also want you to stay in the hospital or center for at least 3 to 6 weeks after your infusion.

Do not use hydroxyurea or medicine to treat HIV for at least 1 month before having a procedure to collect your blood.

Do not use iron chelators for at least 7 days before receiving a cancer medicine and for 6 months after receiving this medicine.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood tests are needed to check for unwanted effects.

Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before starting this medicine. Female patients should use effective birth control during treatment with this medicine and for at least 6 months after the last dose. Male patients who have female partners should use effective birth control during treatment with this medicine and for at least 6 months after the last dose. If you think you have become pregnant while using this medicine, tell your doctor right away.

Betibeglogene autotemcel injection will lower the number of some types of blood cells in your body. Because of this, you may bleed or get infections more easily. To help with these problems, avoid being near people who are sick or have infections. Wash your hands often. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Brush and floss your teeth gently. Be careful when using sharp objects, including razors and fingernail clippers.

This medicine may cause serious allergic reaction, including anaphylaxis, which may be life-threatening and requires immediate medical attention. Check with your doctor right away if you have a rash, itching, dizziness, fainting, fast heartbeat, trouble breathing or swallowing, or chest tightness while you are using this medicine.

Do not donate blood, organs, tissues, or cells during treatment with this medicine.

Before you have any medical tests, tell the medical doctor in charge that you are taking this medicine. The results of some tests may be affected by this medicine.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Black, tarry stools

blurred vision

body aches or pain

chest tightness

chills

confusion

cough or hoarseness

cracked lips

diarrhea

difficulty in swallowing

dizziness

ear congestion

fainting

fever

headache

lightheadedness

loss of voice

lower back or side pain

muscle or bone pain

pain in the arms or legs

painful or difficult urination

pale skin

pounding in the ears

slow or fast heartbeat

sneezing

sore throat

stomach pain

stuffy or runny nose

tender, swollen glands in the neck

trouble breathing

ulcers, sores, or white spots on the lips, tongue, or inside the mouth

unusual bleeding or bruising

unusual tiredness or weakness

Less common

Chest pain

decreased urine output

dilated neck veins

fast, pounding, or irregular heartbeat or pulse

irregular breathing

swelling of the face, fingers, feet, or lower legs

weight gain

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Acid or sour stomach

belching

bleeding gums

bloody nose

constipation

darkening of the skin

decreased appetite

hair loss or thinning of the hair

heartburn

indigestion

lightening of the normal skin color

lightening of treated areas of the dark skin

nausea

stomach discomfort or upset

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.