Pronunciation:

ser-LIP-oh-nase AL-fa

Brand Names:

• Brineura

Dosage Forms:

• Solution

Classifications:

Therapeutic—

Endocrine-Metabolic Agent

Pharmacologic—

Enzyme

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 down 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:

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 performed to date have not demonstrated pediatric-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.

Older adults—

No information is available on the relationship of age to the effects of cerliponase alfa injection in geriatric patients.

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:

• Acute intraventricular access device problems (eg, leakage, extravasation of fluid, failure) or
• Infection of the brain or nerves (eg, meningitis) or
• Infection on or around the device insertion site (eg, cellulitis), acute or uncorrected 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 problems, 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 medical facility. It is injected into the cerebrospinal fluid using an infusion pump system through a tube placed inside your head.

You may be given other medicines (eg, allergy medicines, fever medicines, steroids) 30 to 60 minutes before you receive cerliponase alfa injection to help prevent unwanted effects.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress closely and 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 increase your risk of infections in your brain or nerves (eg, meningitis) or on or around the device insertion site on your head (eg, cellulitis). If you have fever, headache, nausea, vomiting, and a stiff neck or back, or pain, redness, or swelling at the insertion site while receiving this medicine, check with your doctor right away.

This medicine may worsen the condition of patients with a history of heart disease or heart rhythm problems. Check with your doctor right away if you have chest pain or tightness, decreased urine output, dilated neck veins, extreme tiredness or weakness, irregular heartbeat, swelling of the face, fingers, feet, or lower legs, troubled breathing, or weight gain. You might also feel dizzy or faint, or you might have a fast, pounding, or uneven heartbeat.

This medicine may cause serious types of allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child start to have a rash, itching, hoarseness, trouble breathing or swallowing, or any swelling of your hands, face, or mouth after receiving this medicine.

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

chest pain or discomfort

chills

fever

headache

hives, itching, skin rash

hoarseness

itching, pain, redness, or swelling at the insertion site

joint pain, stiffness, or swelling

lightheadedness, dizziness, or fainting

redness of the skin

seizures

slow, fast, or irregular heartbeat

swelling of the eyelids, face, lips, hands, or feet

tightness in the chest

trouble breathing

trouble swallowing

unusual bruising

unusual tiredness

vomiting

Incidence not known

Confusion

drowsiness

general feeling of illness

nausea

severe headache

stiff neck or back

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

Collection of blood under the skin

deep, dark purple bruise

irritability

jitteriness

sweating

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.