Cerliponase alfa

Cerliponase alfa is used to slow down the loss of ability to crawl or walk in children who have symptoms of a rare genetic condition called ceroid lipofuscinosis type 2 disease (CLN2). cerliponase alfa is for children who are at least 3 years old.

Cerliponase alfa may help slow the loss of some physical abilities in children with ceroid lipofuscinosis type 2 disease (CLN2). However, cerliponase alfa is not a cure for this condition.

Cerliponase alfa may also be used for other reasons not mentioned in this medication guide.

Cerliponase alfa is infused directly into the cerebrospinal fluid through a catheter device that is surgically placed in the child's head. This device must be in place for at least 5 to 7 days before your child receives the first dose of cerliponase alfa.

Cerliponase alfa is administered using an infusion pump. The medicine enters the body through a catheter that goes through the skull and into the brain.

Cerliponase alfa is given only by a specific type of healthcare professional. To avoid infection, cerliponase alfa is administered in a sterile medical environment.

This medicine is typically given once every other week. Your child will also receive an infusion of electrolytes after each cerliponase alfa infusion. The whole procedure will take about 4.5 hours to finish.

About 30 to 60 minutes before each cerliponase alfa infusion, your child will receive medication to help prevent certain side effects of this medicine.

Your child's breathing, blood pressure, oxygen levels, and other vital signs will be closely monitored during each infusion. The child's heart function may also need to be checked using an electrocardiograph or ECG (sometimes called an EKG).

In some situations, the child's heart function should be evaluated every 6 months. Be sure to attend all appointments with your child's doctor.

Get emergency medical help if your child shows any signs of an allergic reaction within 24 hours after each infusion. Symptoms may include fever, vomiting, fussiness, hives, trouble breathing, and swelling in the face or throat.

Your child should not be treated with cerliponase alfa if he or she is allergic to it, or if the child has:

• problems with the surgically implanted catheter used to deliver cerliponase alfa;
• any signs of infection on the child's scalp in the area around the implanted catheter; or
• a shunt in the brain (to help drain fluid build-up around the brain).

Tell your doctor if your child has ever had:

• slow heartbeats;
• a heart defect; or
• heart rhythm problems.

Cerliponase alfa is not approved for use by anyone younger than 3 years old.

Get emergency medical help if your child shows signs of an allergic reaction within 24 hours after each infusion. Symptoms may include fever, vomiting, fussiness, hives, trouble breathing, and swelling in the face or throat.

Your child will be closely watched during the cerliponase alfa infusion. Any issues that arise during this time may need additional treatment from healthcare professionals.

Call your child's doctor immediately if you notice:

• swelling, redness, or warmth in the child's scalp;
• bulging or puffiness around the catheter;
• oozing or discharge around the catheter;
• fever with headache, neck stiffness, increased sensitivity to light, vomiting, or drowsiness;
• severe weakness; or
• any changes in behavior, such as a lack of normal responses from your child.

Common side effects of cerliponase alfa may include:

• slow heartbeats, low blood pressure;
• an allergic reaction;
• bruising;
• fever;
• a seizure;
• signs of infection on or around the catheter, such as redness, tenderness, or discharge;
• feeling jittery;
• fussiness;
• vomiting;
• headache; or
• abnormal blood tests or EKGs.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Usual Pediatric Dose for Neuronal Ceroid Lipofuscinosis:

3 years or older: 300 mg by intraventricular infusion once every other week Comments:-Pretreatment with antihistamines with or without antipyretics or corticosteroids should occur 30 to 60 minutes prior to the start of infusion.-Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage, failure, or potential infection. -Prior to each infusion, and when clinically indicated, obtain a sample of CSF for cell count and culture. -Infusion rate 2.5 mL/hr; administer with the B Braun Perfusor Space Infusion Pump System, follow infusion with Intraventricular Electrolytes provided in the administration kit.Use: To slow the loss of ambulation in symptomatic pediatric patients 3 years or older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency

Call your doctor for instructions if you miss an appointment for your child's Cerliponase alfa infusion.

Since cerliponase alfa is given by a healthcare professional in a medical setting, an overdose is unlikely to happen.

Follow your doctor's instructions about any limits on food, drinks, or activities.

Your child's caregivers will take care of and keep track of all medications your child receives during treatment with cerliponase alfa. It is not expected that cerliponase alfa will interact with other medications.

Do not give your child any medications that your doctor has not prescribed. This includes vitamins, minerals, or herbal products.