Pronunciation: brin-nerr-rah
Generic name: cerliponase alfa
Dosage form: injection for intraventricular use
Drug class: Lysosomal enzymes

Medically reviewed by Carmen Pope, BPharm. Last updated on Aug 1, 2024.

What is Brineura?

Brineura (cerliponase alfa) is used to slow the loss of ability to crawl or walk in children and infants with symptoms of a rare genetic condition called ceroid lipofuscinosis type 2 disease (CLN2).

• CLN2 is also known as tripeptidyl peptidase 1 (TPP1) deficiency.
• Administration of Brineura is by a healthcare professional specialized in the administration of medicines via an intraventricular access device (a device that allows medicines to be directly placed into the cerebrospinal fluid).
• Brineura is not a cure for CLN2.

CLN2 disease is a rare condition that damages the brain and nervous system. It happens when the body lacks a special enzyme called TPP1 which helps break down waste proteins in brain cells. Without TPP1, these waste proteins build up, causing problems with movement, thinking, and vision. Brineura (cerliponase alfa) is a special version of the missing TPP1 enzyme which helps break down these proteins. Brineura is called a hydrolytic lysosomal N-terminal tripeptidyl peptidase.

Brineura first gained FDA approval on April 27, 2017. Approval of Brineura was extended to include children under 3 years old with CLN2 on July 24, 2024.

Brineura side effects

Common side effects of Brineura 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

• abnormal blood tests or EKGs.

Serious side effects and warnings

Brineura carries a Boxed Warning for severe hypersensitivity reactions including anaphylaxis.

Severe hypersensitivity reactions including anaphylaxis have occurred with Brineura, usually in people just starting treatment or after an extended period of treatment. Your child will receive Brineura in a healthcare setting with appropriate medical monitoring and support measures. Your child will remain under constant supervision during the Brineura infusion. Any problems the child has during this time may require further treatment by healthcare professionals. 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, difficulty breathing, or swelling in the face or throat.

There is a risk of device-related infections, including meningitis, with Brineura use. Call your child's doctor at once if you notice symptoms of an infection, which may include:

• 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 behavior changes, such as a lack of normal responses from your child.

Infusion-related reactions, low blood pressure, and a slow heart rate may also occur during and following a Brineura infusion. Your healthcare provider will monitor your child for these.

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 the FDA at 1-800-FDA-1088.

Related/similar drugs

Before taking this medicine

Your child should not be treated with Brineura if he or she is allergic to it, or any of the inactive ingredients in the infusion, or if the child has:

• complications with the surgically implanted catheter used to deliver Brineura

• any sign of infection on the child's scalp in the area around the implanted catheter

• 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

• heart rhythm problems.

Brineura is not recommended for babies born earlier than 37 weeks or who weigh less than 2.5 kg (approximately 5.5 pounds).

How is Brineura given?

Brineura is infused directly into 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 Brineura.

• Brineura is given using an infusion pump. The medicine enters the body through a catheter placed through the skull and into the brain.
• Brineura is given only by a certain type of healthcare professional. To prevent infection, this medicine 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 Brineura. The entire procedure will take about 4.5 hours to complete.

• About 30 to 60 minutes before each Brineura 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.

Brineura dosing

The dosage of Brineura and the infusion rate depends on a child's age.

What happens if I miss a dose?

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

What happens if I overdose?

Since Brineura is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while receiving Brineura?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect Brineura?

Your child's caregivers will manage and monitor all medications given to the child during treatment with Brineura. A drug interaction between Brineura and other medications is not expected to occur.

Do not give any medications to your child that have not been prescribed by your doctor. This includes vitamins, minerals, or herbal products.

Brineura ingredients

Active ingredient: cerliponase alfa (a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase).
Each vial of Brineura provides 5 mL of solution containing 150 mg cerliponase alfa.

Inactive ingredients: Both Brineura and intraventricular electrolytes are formulated with the following excipients: calcium chloride dihydrate (1.05 mg); magnesium chloride hexahydrate (0.8 mg); potassium chloride (1.1 mg); sodium chloride (43.85 mg); sodium phosphate, dibasic, heptahydrate (0.55 mg); sodium phosphate, monobasic, monohydrate (0.4 mg); and water for Injection, USP.

• The pH of the solution is between 6.2 to 6.8 for Brineura, and between 6.0 to 7.0 for intraventricular electrolytes Injection.
• Each vial contains: sodium: 0.76 mEq, and potassium: 0.015 mEq.
• Each vial of Intraventricular Electrolytes Injection provides
  5 mL of solution.

Who makes Brineura?

BioMarin Pharmaceutical Inc. makes Brineura.

Brineura Biosimilars

Biosimilar and interchangeable products are biological products that are highly similar to and have no clinically meaningful differences from the reference product.

Reference products

These are biological products that have already been approved by the FDA, against which biosimilar products are compared. There is 1 for Brineura.

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Further information

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