Pronunciation:

el-a-DOE-ka-jeen ex-ue-PAR-voe-vek - tneq

Uses of This Medicine:

Eladocagene exuparvovec-tneq injection is used to treat aromatic L-amino acid decarboxylase (AADC) deficiency. Your doctor will perform a test before you receive this medicine.

This medicine is to be given only by or under the direct supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of eladocagene exuparvovec-tneq injection in children younger than 16 months of age. Safety and efficacy have not been established.

Older adults—

Appropriate studies on the relationship of age to the effects of eladocagene exuparvovec-tneq injection have not been performed in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Skull maturity, not reached—Should not be used in patients with this condition.

Proper Use of This Medicine:

A doctor will give you this medicine in a medical facility. It is injected into the putamen part of the brain using an infusion pump system through a tube placed inside your head.

Precautions While Using This Medicine:

It is very important that your doctor check your progress closely and at regular visits to make sure this medicine is working properly. Blood tests will be needed to check for unwanted effects.

If you can get pregnant, your doctor may do tests to make sure you are not pregnant before receiving this medicine.

This medicine may cause complications during procedure (eg, cardiac arrest, respiratory arrest, cerebrospinal fluid leak, intracranial bleeding, neuroinflammation, acute infarction, and infection). Check with your doctor right away if you have blurred vision, confusion, fever or chills, cough or hoarseness, headache, lower back or side pain, nausea and vomiting, numbness or tingling in the face, arms, or legs, no blood pressure or pulse, no breathing, painful or difficult urination, stopping of the heart, trouble speaking or walking, unconsciousness, or weakness.

This medicine may cause uncontrolled sudden movements (dyskinesia) or make such movements you already have worse or more frequent. Check with your doctor right away if you have twitching, twisting, uncontrolled repetitive movements of the tongue, lips, face, arms, or legs.

Do not donate blood, organs, tissues, or cells for transplant after treatment.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Blurred vision

bone pain

confusion

decreased urine

dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position

drowsiness

dry mouth

increased thirst

irregular heartbeat

loss of appetite

mood changes

muscle pain, spasms, or cramps

nausea or vomiting

no blood pressure or pulse

no breathing

numbness or tingling in the hands, feet, or lips

pale skin

seizures

stopping of the heart

sweating

trouble breathing

trembling

twitching, twisting, uncontrolled repetitive movements of the tongue, lips, face, arms, or legs

unconsciousness

unusual bleeding or bruising

unusual tiredness or weakness

Incidence not known

Cough or hoarseness

fever or chills

headache

lower back or side pain

painful or difficult urination

trouble speaking or walking

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Increased watering of mouth

trouble sleeping

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.