Elivaldogene autotemcel is used to slow nerve damage in boys 4 to 17 years old with early, active cerebral adrenoleukodystrophy (CALD), a genetic disease that may damage the brain.

Elivaldogene autotemcel may also be used for purposes not listed in this medication guide.

Call your child's doctor at once if your child has bruising or bleeding, blood in your urine or stool, severe headache, stomach or back pain, fever, swollen glands, tiredness, or if you are vomiting or coughing up blood.

Follow all directions on the prescription label and read all medication guides or instruction sheets.

Elivaldogene autotemcel is injected into a vein by a healthcare provider. Your child will be given other medicines as part of the treatment with elivaldogene autotemcel.

After elivaldogene autotemcel injection, your child may stay in the hospital for approximately 2 months to monitor the recovery. Your child's doctor will determine when your child can go home.

This medicine may cause false results on a HIV (human immunodeficiency virus) test. Tell the laboratory staff that your child uses elivaldogene autotemcel.

Blood will need to be tested often.

Elivaldogene autotemcel may increase the risk of cancer. Ask a healthcare provider about your child's risk.

Usual Pediatric Dose for Cerebral Adrenoleukodystrophy:

Minimum recommended dose: 5.0*10(6) CD34+ cells/kg as a single dose for intravenous infusion

Comments:
-This indication is approved under accelerated approval based on 24-month major functional disability (MFD)-free survival.
-Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
-The eligibility for early, active cerebral adrenoleukodystrophy (CALD) is defined as asymptomatic or mildly symptomatic (neurologic function score, NFS 1 or less) patients who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.
-Dose calculation is based on individual body weight prior to first apheresis. Refer to manufacturer product information for detailed calculation.
-This drug product is not indicated for prevention of development or treatment of adrenal insufficiency due to adrenoleukodystrophy.
-This drug product has not been studied in patients with cerebral adrenoleukodystrophy (CALD) secondary to head trauma.

Use: For boys 4 to 17 years of age with early, active cerebral adrenoleukodystrophy

Tell the doctor if your child has ever had:

• liver or kidney disease;

• an allergy to dimethyl sulfoxide (DMSO, a preserving agent); or

• if your child has an active infection.

It may be harder for your child to get a female pregnant while using elivaldogene autotemcel. Your child should still use birth control to prevent pregnancy until at least 6 months after administration of elivaldogene autotemcel.

Call your child's doctor for instructions if your child misses an appointment for the elivaldogene autotemcel injection.

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

Your child should avoid donating blood, organs, tissues, or cells.

Get emergency medical help if your child has signs of an allergic reaction: hives; difficult breathing; swelling of the face, lips, tongue, or throat.

Call the doctor at once if your child has:

• signs of blood cancer--bruising or bleeding, blood in the urine or stool, severe headache, stomach or back pain, swollen glands, or vomiting or coughing up blood;

• signs of infection--fever, chills, sore throat, body aches, unusual tiredness, loss of appetite, bruising or bleeding; or

• low blood cell counts--mouth sores, skin sores, easy bruising, unusual bleeding, pale skin, cold hands and feet, feeling light-headed or short of breath.

Common side effects of elivaldogene autotemcel may include:

• fever, chills, sore throat, body aches, unusual tiredness, loss of appetite, bruising or bleeding;

• skin sores, easy bruising, pale skin, cold hands and feet, feeling light-headed or short of breath;

• leg cramps, irregular heartbeats, fluttering in the chest, increased thirst or urination, numbness or tingling, muscle weakness or limp feeling;

• sores or white patches in or around the mouth, trouble swallowing or talking, dry mouth, bad breath, altered sense of taste;

• nausea, vomiting, loss of appetite, stomach pain, constipation, diarrhea;

• hair loss;

• headache; or

• rash.

This is not a complete list of side effects and others may occur. Call your child's doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Tell the doctor about all your child's other medicines, especially:

• HIV/AIDS medications.

This list is not complete. Other drugs may affect elivaldogene autotemcel, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.