Eladocagene exuparvovec-tneq Injection

WHY is this medicine prescribed?

Eladocagene exuparvovec-tneq is used to treat L-amino acid decarboxylase (AADC) deficiency (genetic disease that alters the production of signals that allow cells in the nervous system to communicate with each other). Eladocagene exuparvovec-tneq is in a class of medications called gene therapy. It works by improving production of signals needed for cells in the nervous system to communicate.

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?

Eladocagene exuparvovec-tneq comes as a suspension (liquid) to be given intraputaminally (into a region of the brain) during a surgical procedure at a hospital that is able to perform these procedures. Your doctor will tell you what to expect before, during and after the procedure to receive eladocagene exuparvovec-tneq. The surgical procedure to give eladocagene exuparvovec-tneq may be associated with complications and so your doctor will monitor you closely in the hospital for a period of time.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

What SPECIAL PRECAUTIONS should I follow?

Before receiving eladocagene exuparvovec-tneq,

• tell your doctor and pharmacist if you are allergic to eladocagene exuparvovec-tneq, any other medications, or any of the ingredients in eladocagene exuparvovec-tneq injection. Ask your pharmacist for a list of the ingredients.

• some medications should not be taken with eladocagene exuparvovec-tneq. Make sure you have discussed any medications you are currently taking or plan to take before starting eladocagene exuparvovec-tneq injection with your doctor and pharmacist. Before starting, stopping, or changing any medications while taking eladocagene exuparvovec-tneq injection, please get the advice of your doctor or pharmacist.

• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take.

• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. Your doctor may have you take a pregnancy test prior to receiving eladocagene exuparvovec-tneq. If you become pregnant after receiving eladocagene exuparvovec-tneq, call your doctor.

What SPECIAL DIETARY instructions should I follow?

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

What SIDE EFFECTS can this medicine cause?

Eladacogene exuparvovac-tneq may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• fever
• excess salivation
• trouble falling or staying asleep
• feeling dizzy or lightheaded
• feeling tired, pale skin, shortness of breath, headache, cold hands or feet, brittle nails or hair loss
• muscle weakness or cramping
• muscle weakness, spasms or tremors; nausea, vomiting or loss of appetite; abnormal eye movements

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately:

• involuntary movements of face, arm, leg or body such as fidgeting, writhing, wriggling, head bobbing or body swaying

Eladocagene exuparvovec-tneq 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 (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor may order certain lab tests to check your body's response to eladocagene exuparvovec-tneq.

Eladocagene exuparvovec-tneq is made from adeno-associated virus (small viruses that can infect humans). Vector shedding (release of parts of virus from the patient's body) can occur from 3 weeks after receiving eladocagene exuparvovec-tneq. Be sure to wash hands frequently and use gloves for dressing changes and handling of any waste material (e.g. blood, nasal secretions, urine, stool). Put any waste materials that have come into contact with body fluids in sealed bags before disposing of it. Do not donate blood, organs, tissues or cells for transplantation.

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.