Generic Name: cerliponase alfa (ser LIP oh nase AL fa)
Brand Name: Brineura
Medically reviewed: July 25, 2017
What is cerliponase alfa?
Cerliponase alfa contains an enzyme that occurs naturally in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Cerliponase alfa replaces this missing enzyme in such people.
Cerliponase alfa is used to slow the loss of ability to crawl or walk in children with symptoms of a rare genetic condition called ceroid lipofuscinosis type 2 disease (CLN2), also called Batten disease. This medicine is for use in children who are at least 3 years old.
Batten disease is a rare genetic condition in which a person lacks the enzyme needed to break down certain materials in the body. These materials can build up in brain cells and cause problems with movement and coordination, delayed speech development, vision problems, seizures, and dementia.
Cerliponase alfa may help slow the loss of certain physical abilities in children with Batten disease. However, cerliponase alfa is not a cure for this condition.
Cerliponase alfa may also be used for purposes not listed in this medication guide.
Your child will receive cerliponase alfa in a medical setting, and will remain under constant supervision while this medicine is infused.
Before taking this medicine
Your child should not be treated with cerliponase alfa if he or she is allergic to it, or if the child has:
complications with the surgically implanted catheter used to deliver this medicine; or
a shunt in the brain (to help drain fluid build-up around the brain).
To make sure cerliponase alfa is safe for your child, tell your doctor if the child has ever had:
a heart defect; or
heart rhythm problems.
Cerliponase alfa is not approved for use by anyone younger than 3 years old.
How is cerliponase alfa given?
Cerliponase alfa is infused directly in the cerebrospinal fluid through a catheter device that is surgically implanted in the child's head. This device must be in place at least 5 to 7 days before your child gets his or her first dose of cerliponase alfa.
Cerliponase alfa is given using an infusion pump. The medicine enters the body through a catheter placed through the skull and into the brain.
Cerliponase alfa is given only by a certain type of healthcare professional. To prevent infection, cerliponase alfa is given in a sterile medical setting.
This medicine is usually given once every other week. Your child will also receive an infusion of electrolytes after each infusion of cerliponase alfa. The entire procedure will take about 4.5 hours to complete.
About 30 to 60 minutes before each cerliponase alfa infusion, your child will be given medication to prevent certain side effects of this medicine.
Your child's breathing, blood pressure, oxygen levels, and other vital signs will be watched closely during each infusion. The child's heart function may also need to be monitored using an electrocardiograph or ECG (sometimes called an EKG).
In some cases, the child's heart function should be checked every 6 months. Be sure to keep all appointments with your child's doctor.
What happens if I miss a dose?
Call your doctor for instructions if you miss an appointment for your child's cerliponase alfa infusion.
What happens if I overdose?
Since this medicine is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.
What should I avoid while receiving cerliponase alfa?
Follow your doctor's instructions about any restrictions on food, beverages, or activity.
Cerliponase alfa side effects
Get emergency medical help if your child has signs of an allergic reaction within 24 hours after each infusion. Symptoms may include fever, vomiting, fussiness, hives, difficult breathing, and swelling in the face or throat.
Your child will remain under constant supervision during the cerliponase alfa infusion. Any problems the child has during this time may require further treatment by healthcare professionals.
Call your child's doctor at once if you notice:
swelling, redness, or warmth in the child's scalp;
bulging or puffiness around the catheter; or
any behavior changes, such as a lack of normal responses from your child.
Common side effects may include:
slow heartbeats, low blood pressure;
fussy or irritable behavior;
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.
See also: Side effects (in more detail)
Cerliponase alfa dosing information
Usual Pediatric Dose for Neuronal Ceroid Lipofuscinosis:
3 years or older: 300 mg by intraventricular infusion once every other week
-Pretreatment with antihistamines with or without antipyretics or corticosteroids should occur 30 to 60 minutes prior to the start of infusion.
-This drug should be administered first, followed by the intraventricular electrolytes using the provided 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
What other drugs will affect cerliponase alfa?
Your child's caregivers will manage and monitor all medications given to the child during treatment with this medicine. A drug interaction between cerliponase alfa and other medications is not expected to occur.
Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.
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More about cerliponase alfa
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- Drug class: lysosomal enzymes
Other brands: Brineura