Cerliponase alfa (Intracerebroventricular route)
Uses of This Medicine:
Cerliponase alfa injection is used to treat late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) or tripeptidyl peptidase 1 (TPP1) deficiency. It helps slow the loss of walking ability in children 3 years of age and older.
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:
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.
Appropriate studies performed to date have not demonstrated pediatrics-specific problems that would limit the usefulness of cerliponase alfa injection in children 3 years of age and older. However, safety and efficacy have not been established in children younger than 3 years of age.
No information is available on the relationship of age to the effects of cerliponase alfa injection in geriatric patients.
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.
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.
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:
- Acute intraventricular access device problems (eg, leakage, failure, device-related infection) or
- Ventriculoperitoneal shunt (a medical device that relieves pressure on the brain)—Should not be used in patients with these conditions.
- Bradycardia (slow heartbeat), history of or
- Heart disease, history of or
- Heart rhythm problem, history of or
- Hypotension (low blood pressure), history of—Use with caution. May make these conditions worse.
Proper Use of This Medicine:
A doctor or other trained health professional will give you this medicine in a clinic or hospital. This medicine is given into the cerebrospinal fluid using an infusion pump system through a tube placed inside the head.
You may be given other medicines (eg, allergy medicines, fever medicines, steroids) at least 30 to 60 minutes before you receive cerliponase alfa injection to help prevent infusion reactions.
Precautions While Using This Medicine:
It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood tests may be needed to check for unwanted effects.
This medicine may cause serious types of allergic reactions, including infusion reaction and anaphylaxis. These can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child start to have cough, trouble breathing, hives, itching, or skin rash, lightheadedness, dizziness, or fainting, tightness in the chest, or swelling of the face or lips.
Side Effects of This Medicine:
- More common
- Blurred vision
- chest pain or discomfort
- hives, itching, or rash
- lightheadedness, dizziness, or fainting
- slow or irregular heartbeat
- swelling of the face, tongue, or throat
- trouble breathing
- trouble swallowing
- unusual bruising
- unusual tiredness
- More common
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:
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:
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.
Last Updated: 9/4/2017