Pronunciation:

ex-a-GAM-gloe-jeen aw-toe-TEM-sel

Brand Names:

• Casgevy

Dosage Forms:

• Suspension

Classifications:

Therapeutic—

Blood Modifier Agent

Uses of This Medicine:

Exagamglogene autotemcel injection is used to treat sickle cell disease (red blood cell disease) in patients who have frequent vaso-occlusive crisis. This medicine is made from human blood stem cells. It is used to increase the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin) which helps improve the production and formation of red blood cells.

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 exagamglogene autotemcel injection in children younger than 12 years of age. Safety and efficacy have not been established.

Older adults—

Appropriate studies on the relationship of age to the effects of exagamglogene 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.

• Crizanlizumab
• Deferasirox
• Deferiprone
• Deferoxamine Mesylate
• Hydroxyurea
• Voxelotor

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:

• Allergy to dimethyl sulfoxide (DMSO), history of—Use with caution. May increase the risk of an allergic reaction.

• Kidney disease or
• Liver disease—Use with caution. This medicine has not been studied in patients with these conditions.

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 usually comes with patient information leaflet. Read and follow the instructions carefully and make sure you understand them before receiving this medicine.

You may also receive other medicines (eg, fever medicine or allergy 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 4 to 6 weeks after your infusion.

Do not use crizanlizumab, hydroxyurea, and voxelotor for at least 8 weeks and an iron chelator for at least 7 days before having a procedure to collect your blood.

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.

If you are a woman who can get pregnant, your doctor may do tests to make sure you are not pregnant before having a procedure to collect your blood. Female patients and male patients with female partners should use an effective form of birth control before having a procedure to collect your blood and for at least 6 months after the last dose.

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.

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

bleeding gums

blood in the urine or stools

chest pain

chills

cough

fever

gaseous stomach pain

lower back or side pain

painful or difficult urination

pale skin

pinpoint red spots on the skin

sore throat

stomach fullness

swollen glands

trouble breathing

ulcers, sores, or white spots in the mouth

unusual bleeding or bruising

unusual tiredness or weakness

yellow eyes or skin

Less common

Back pain

bloated stomach

chest tightness

fast heartbeat

flushing

headache

nausea and vomiting

pain and fullness in the right upper abdomen

weight gain

Incidence not known

Difficulty swallowing

dizziness

hives, itching, skin rash

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

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

Cracked lips

decreased appetite

diarrhea

muscle or bone pain

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.