Cerliponase alfa Injection

Cerliponase alfa may cause hypersensitivity (severe allergic) reactions, including life-threatening reactions such as anaphylaxis, during and for 24 hours after you complete your infusion. Your doctor or nurse will monitor you very closely during your infusion. This type of reaction may happen early in your treatment or after you have received many doses. If you experience any of the following symptoms after receiving cerliponase alfa, you should seek emergency medical care: rash; itching; hives; swelling of the face, throat, tongue, lips, and eyes; difficulty breathing or swallowing; or wheezing. Your doctor may give you medicines 30 to 60 minutes prior to each infusion to reduce the chance of these events occurring.

Cerliponase alfa is used to slow the loss of being able to crawl or walk in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease; an inherited disease of the nervous system), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Cerliponase alfa is in a class of medications called enzymes. It works by replacing the lack of TPP1 in the nervous system.

Cerliponase alfa comes as a solution (liquid) to be diluted and infused slowly over several hours into an access device that was previously placed into the brain by a surgical procedure. It is usually given every 2 weeks (every other week) in a doctor's office or healthcare facility.

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

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

Before receiving cerliponase alfa,

• tell your doctor and pharmacist if you are allergic to cerliponase alfa, any other medications, or any of the ingredients in cerliponase alfa 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 an infection near your access device (redness, tenderness, discharge) or if you have leaking from the device site. Your doctor will probably tell you that you should not receive cerliponase alfa.
• tell your doctor if you have or have ever had an irregular heartbeat or other heart problems.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding.

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

If you miss the appointment to receive your cerliponase alfa dose, you must call your doctor right away. If you miss one or more doses, you should restart cerliponase alfa treatment as soon as possible, and then will return to the 2-week interval between treatments.

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

• vomiting
• bruising
• headache
• irritability
• feeling jittery

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

• fever
• seizures
• slow or irregular heartbeat
• dizziness or faintness

Cerliponase alfa 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).

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to cerliponase alfa.

Ask your pharmacist any questions you have about cerliponase alfa.

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.

• Brineura^®