Elivaldogene Autotemcel (el-i-VAL-doe-jeen aw-toe-TEM-sel)

Treats early, active cerebral adrenoleukodystrophy (CALD).

Brand Name(s):

Skysona

When This Medicine Should Not Be Used:

How to Use This Medicine:

Injectable

• Your doctor will prescribe your dose and schedule. This medicine 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 60 minutes.
• A nurse or other health provider will give you this medicine.
• You may also receive other medicines (including cancer medicine, fever medicine, allergy medicine, or medicine to prevent nausea or vomiting) 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 2 months after your infusion.
• This medicine should come with a Medication Guide. Ask your pharmacist for a copy if you do not have one.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

• Do not use medicine to treat HIV for at least 1 month before having a procedure to collect your blood.
• This medicine may interfere with vaccines. Ask your doctor before you get a flu shot or any other vaccines. You should not receive a live vaccine for at least 6 weeks before the start of treatment, during treatment, and until recovery following the last cycle of this medicine.

Warnings While Using This Medicine:

• This medicine is used in boys 4 to 17 years of age.
• 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.
• Tell your doctor if you have kidney disease, liver disease, any type of infection, or a history of dimethyl sulfoxide (DMSO) allergy.
• This medicine may cause the following problems:
  • Blood cancer, including myelodysplastic syndrome (MDS)
  • Increased risk of serious infections, which may be life-threatening
• This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.
• Do not donate blood, organs, tissues, or cells during treatment with this medicine.
• Tell any doctor or dentist who treats you that you are using this medicine. This medicine may affect certain medical test results.
• Your doctor will need to check your blood every 6 months for at least 15 years while you are using this medicine. Be sure to keep all appointments.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

• Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
• Change in how much or how often you urinate, painful or difficult urination
• Fever, chills, cough, sore throat, body aches
• Seizures
• Unusual bleeding, bruising, or weakness

If you notice these less serious side effects, talk with your doctor:

• Constipation, diarrhea, nausea, vomiting, stomach pain, loss of appetite
• Hair loss
• Headache
• Sores or white patches on your lips, mouth, or throat

If you notice other side effects that you think are caused by this medicine, tell your doctor

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