Elivaldogene autotemcel Injection

(el" i val' doe jeen aw" toe tem' sel)

Brand Name(s): , Skysona^®

---

IMPORTANT WARNING:

Elivaldogene autotemcel injection may increase the risk that you will develop life-threatening blood cancer. Your blood will be monitored at least every 6 months for at least 15 years after your treatment with elivaldogene autotemcel injection. If you experience any of the following symptoms, call your doctor immediately: unusual bruising or bleeding; nose bleed; severe headache; blood in urine, stool, or vomit; coughing up blood; or unusual abdominal or back pain. Talk to your doctor about the risks of taking this medication.

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

Your doctor or pharmacist will give you the manufacturer's patient information sheet (Medication Guide) when you begin treatment with elivaldogene autotemcel injection. Read the information carefully and ask your doctor or pharmacist if you have any questions. You can also visit the Food and Drug Administration (FDA) website ([WEB]) or the manufacturer's website to obtain the Medication Guide.

WHY is this medicine prescribed?

Elivaldogene autotemcel injection is used to slow the progression of brain damage and slow the decline of nerve function in males 4–17 years of age with early, active cerebral adrenoleukodystrophy (CALD; an inherited condition in which fatty acids build up in the brain, resulting in nerve and brain damage). Elivaldogene autotemcel injection is in a class of medications called autologous cellular immunotherapy, a type of medication prepared using cells from the patient's own blood. It works by helping your body to break down the fatty acids and slow the progression of nerve and brain damage.

Are there OTHER USES for this medicine?

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

HOW should this medicine be used?

Elivaldogene autotemcel injection comes as a suspension (liquid) to be injected intravenously (into a vein) by a doctor or nurse in a hospital. Elivaldogene autotemcel injection is given as a one-time dose, which is contained in 1 or 2 infusion bags. Each infusion bag is usually given over no more than 60 minutes. Before you receive your elivaldogene autotemcel dose, your doctor or nurse will administer other chemotherapy medications to prepare your body for elivaldogene autotemcel.

Before your dose of elivaldogene 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 elivaldogene autotemcel injection. It will take about 51–65 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.

Elivaldogene autotemcel injection may cause nausea and vomiting during treatment with the medication. Your doctor may give you other medications to help prevent nausea and vomiting.

You will have to stay in the hospital for about 2 months after you receive your elivaldogene 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.

What SPECIAL PRECAUTIONS should I follow?

Before receiving elivaldogene autotemcel injection,

• tell your doctor and pharmacist if you are allergic to elivaldogene autotemcel, dimethyl sulfoxide (DMSO), any other medications, or any of the ingredients in elivaldogene autotemcel injection. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what 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 human immunodeficiency virus (HIV), or kidney or liver problems.
• you should know that elivaldogene autotemcel injection is only for use in males. If you are male, you and your female partner should use birth control to prevent pregnancy during your treatment and for at least 6 months after you receive elivaldogene autotemcel injection. Talk to your doctor about birth control methods that will work for you. If your partner becomes pregnant while you are receiving elivaldogene autotemcel injection, call your doctor.
• you should know that elivaldogene autotemcel injection may decrease fertility in men. Talk to your doctor about the risks of receiving elivaldogene autotemcel injection.
• do not donate blood, organs, tissues, or cells for transplantation after you receive elivaldogene autotemcel injection.
• check with your doctor to see if you need to receive any vaccinations. Do not have any vaccinations without talking to your doctor for at least 6 weeks before starting chemotherapy, during your elivaldogene autotemcel treatment, and until your doctor tells you that your immune system has recovered.

What SPECIAL DIETARY instructions should I follow?

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

What SIDE EFFECTS can this medicine cause?

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

• nausea
• vomiting
• constipation
• diarrhea
• stomach pain
• decreased appetite
• mouth sores
• blurred vision
• headache
• hair loss
• darkening of the skin

Some side effects can be serious. If you experience any of these symptoms or those listed in the IMPORTANT WARNING section, call your doctor immediately or get emergency medical treatment:

• rash; itching; hives; swelling of your face, mouth, tongue, or throat; chest pain; fast heartbeat; or difficulty swallowing or breathing
• unusual bruising or bleeding; nose bleed; severe headache; blood in urine, stool, or vomit; coughing up blood; or unusual abdominal or back pain
• fever, sore throat, swollen glands, ongoing cough and congestion, or other signs of infection
• weakness, feeling tired, or shortness of breath
• seizure

Elivaldogene autotemcel injection may cause other side effects. Call your doctor if you have any unusual problems while taking 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 ([WEB]) or by phone (1-800-332-1088).

What should I do in case of OVERDOSE?

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

What OTHER INFORMATION should I know?

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

---

This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.

The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.

AHFS^® Patient Medication Information™. © Copyright, 2023. The American Society of Health-System Pharmacists^®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.

Selected Revisions: November 15, 2022.