Eladocagene Exuparvovec Injection

What is this medication?

ELADOCAGENE EXUPARVOVEC (EL a DOE ka jeen EX ue PAR voe vek) treats aromatic amino acid decarboxylase (AADC) deficiency. It works by helping nerve cells in the brain make a missing enzyme. This helps the cells make substances that let them send signals to each other. This decreases 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.

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

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

• An unusual or allergic reaction to eladocagene exuparvovec, other medications, foods, dyes, or preservatives
• Pregnant or trying to get pregnant
• Breastfeeding

How should I use this medication?

This medication is infused into the area of the brain called the putamen. It is given by your care team in a hospital or clinic setting.

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?

Interactions have not been studied.

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.

After gene therapy, it is important to avoid contact with the treated person's body fluids for 3 weeks. Body fluids include saliva, urine, stool, blood, and semen. By avoiding contact, the gene therapy will not affect others by accident. Wash your hands with soap and water after coming in contact with body fluids and before eating. Use disposable gloves. Place items that have body fluids on them, such as diapers, in a sealed bag. Get rid of the bag in the regular trash.

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

Talk to your care team if you may be pregnant. This medication has not been well studied during pregnancy. You will need a negative pregnancy test before starting this medication.

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
• Bleeding in the brain—severe headache, stiff neck, confusion, dizziness, change in vision, numbness or weakness of the face, arm, or leg, trouble speaking, trouble walking, vomiting
• Heart rhythm changes—fast or irregular heartbeat, dizziness, feeling faint or lightheaded, chest pain, trouble breathing
• Infection—fever, chills, cough, or sore throat
• New or worsening uncontrolled and repetitive movements of the face, mouth, or upper body
• Shortness of breath or trouble breathing
• Stroke—sudden numbness or weakness of the face, arm, or leg, trouble speaking, confusion, trouble walking, loss of balance or coordination, dizziness, severe headache, change in vision

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

• Excessive drooling
• Fever
• Trouble sleeping

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.