Atidarsagene Autotemcel Injection

What is this medication?

ATIDARSAGENE AUTOTEMCEL (AT i DAR sa jeen AW toe TEM sel) treats metachromatic leukodystrophy (MLD). It works by helping your body make the enzyme that breaks down the buildup of lipids on nerve cells. This protects the nerve cells and may slow or stop worsening symptoms. It is a gene therapy.

This medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

COMMON BRAND NAME(S): Lenmeldy

What should I tell my care team before I take this medication?

They need to know if you have any of these conditions:

• History of blood clots
• HIV or AIDs
• Infection
• Recent or upcoming vaccine
• An unusual or allergic reaction to atidarsagene autotemcel, other medications, foods, dyes, or preservatives
• Pregnant or trying to get pregnant
• Breastfeeding

How should I use this medication?

This medication is infused into a vein. It is given by your care team in a hospital or clinic setting.

This therapy uses your own stem cells. Your care team will give you other medications as part of your treatment. Before therapy, your care team will give you a medication that helps move your stem cells from your bone marrow to the blood stream, so they can collect them. This is called mobilization. These cells will be used to make your treatment. Shortly before you receive treatment, you will receive another medication that helps prepare your body for the stem cell transplant. This is called conditioning.

Talk to your care team about the use of this medication in children. While it may be given to children for selected conditions, precautions do apply.

Overdosage: If you think you have taken too much of this medicine contact a poison control center or emergency room at once.

NOTE: This medicine is only for you. Do not share this medicine with others.

What if I miss a dose?

This does not apply. This medication is not for regular use.

What may interact with this medication?

• Antivirals for HIV
• Vaccines

This list may not describe all possible interactions. Give your health care provider a list of all the medicines, herbs, non-prescription drugs, or dietary supplements you use. Also tell them if you smoke, drink alcohol, or use illegal drugs. Some items may interact with your medicine.

What should I watch for while using this medication?

Your condition will be monitored carefully while you are receiving this medication. It is important for you to have regular check-ups after this treatment.

You may need blood work done while you are taking this medication.

Do not donate blood, organs, tissues, or cells any time after receiving this treatment.

Talk to your care team about your risk of cancer. You may be more at risk for certain types of cancer if you take this medication.

This medication may increase your risk of getting an infection. Call your care team for advice if you get a fever, chills, sore throat, or other symptoms of a cold or flu. Do not treat yourself. Try to avoid being around people who are sick.

If you need an HIV test, talk to your care team about which test to use. This medication may cause a false-positive result if certain tests are used.

Talk to your care team if you or your partner may be pregnant. You will need a negative pregnancy test before starting treatment. Contraception is recommended during treatment and for at least 6 months after treatment. Your care team can help you find the option that works for you.

If your partner can get pregnant, use a condom during sex during treatment and for at least 6 months after treatment.

Talk to your care team before breastfeeding.

The conditioning medication may cause infertility. Talk to your care team if you are concerned about your fertility.

What side effects may I notice from receiving this medication?

Side effects that you should report to your care team as soon as possible:

• Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
• Blood clot—pain, swelling, or warmth in the leg, shortness of breath, chest pain
• Dizziness, loss of balance or coordination, confusion or trouble speaking
• Infection—fever, chills, cough, sore throat, wounds that don't heal, pain or trouble when passing urine, general feeling of discomfort or being unwell
• Liver injury—right upper belly pain, loss of appetite, nausea, light-colored stool, dark yellow or brown urine, yellowing skin or eyes, unusual weakness or fatigue
• Unusual bruising or bleeding

Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):

• Fever
• Pain, redness, or swelling with sores inside the mouth or throat
• Skin rash

This list may not describe all possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Where should I keep my medication?

This medication is given in a hospital or clinic. It will not be stored at home.

NOTE: This sheet is a summary. It may not cover all possible information. If you have questions about this medicine, talk to your doctor, pharmacist, or health care provider.