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Brineura® (cerliponase alfa) is the only treatment that directly addresses the cause of CLN2 disease by helping to replace the TPP1 enzyme1

What is Brineura?

Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.1

CLN2 disease is a type of lysosomal storage disorder that affects cells in the brain. Lysosomes are in every cell and contain enzymes that break down materials in the cell. One of these enzymes is called TPP1, which is missing or not working properly in children with CLN2 disease.2

The TPP1 enzyme is missing or not working properly in children with CLN2 disease. This leads to a buildup of storage materials in their lysosomes, associated with cell damage in the brain.2-4

When Brineura is delivered to a child with CLN2 disease, it helps replace the missing or malfunctioning TPP1 enzyme.1

Learn more about CLN2 disease

How is Brineura administered to my child?

Brineura is a type of treatment called enzyme replacement therapy (ERT). It’s administered through intraventricular infusion—a method that allows Brineura to be directly delivered into the fluid surrounding the brain, known as the cerebrospinal fluid.1

Before starting Brineura, your child will need to have an intraventricular access device surgically implanted just below their scalp. This is an established procedure in pediatric neurology, and allows direct delivery of Brineura into a ventricle in the brain. The access device is about the size of a penny. It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.1,5

Brineura treatment will take approximately 2 to 4.5 hours, depending on the dose and volume administered, every other week. You’ll work with your healthcare team to schedule treatments.1

Your healthcare team will let you know how to prepare your child for infusion, and what to expect during this procedure. Your child will be monitored before, during, and after the infusion, and may receive medications to reduce the risk of hypersensitivity reactions.1

Your doctor may also take the steps to minimize the risk of device-related infections at every treatment. You may be asked to wear a mask and the number of people in the room will be limited. Ask your team who and what is allowed in the room.1

If you see signs of infection, such as swelling or reddening of the skin, contact your healthcare team immediately.1

Matty’s infusion day

Matty is a patient with CLN2 disease. He’s been on treatment with Brineura since 2015.

After the first few infusions we became more comfortable. The initial surgery for the port was scary, so we felt like that was the first hurdle to jump. Once Matty had a few infusions, we became more comfortable with the process.

—Joe, Matty’s dad
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Treatment

References:

  1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.
  2. Mole SE, Williams RE, Goebel HH. Correlations between genotype, ultrastructural morphology and clinical phenotype in the neuronal ceroid lipofuscinoses. Neurogenetics. 2005;6:107-126.
  3. Haltia M. The neuronal ceroid-lipofuscinoses: from past to present. Biochim Biophys Acta. 2006;1762:850-856.
  4. Schulz A, Kohlschütter A, Mink J, Simonati A, Williams R. NCL diseases – clinical perspectives. Biochim Biophys Acta. 2013;1832:1801-1806.
  5. Cohen-Pfeffer JL, Gururangan S, Lester T, et al. Intracerebroventricular delivery as a safe, long-term route of drug administration. Pediatr Neurol. 2017;67:23-35.

Important Safety Information

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur

What is the most important safety information I should know about Brineura?

Severe and life-threatening allergic reactions, including anaphylaxis, can occur during Brineura infusions and up to 24 hours after infusion. These reactions can occur in people receiving Brineura for the first time or in people who have previously received Brineura without having an allergic reaction. Your child’s doctor should ensure appropriately trained personnel and equipment for emergency resuscitation (including epinephrine and other emergency medicines) are readily available during your child’s Brineura infusion.

Your child’s doctor will tell you about the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and when to seek immediate medical care. Signs of anaphylaxis can include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. If a severe allergic reaction (e.g., anaphylaxis) occurs during infusion, the infusion should be stopped immediately, and your child should receive medical attention. Contact your child’s doctor or get medical help right away if your child develops any severe symptoms after infusion.

If anaphylaxis occurs, you and your child’s healthcare providers should consider the risks and benefits of readministration of Brineura. If the decision is made to readminister Brineura after the occurrence of anaphylaxis, the healthcare providers should ensure appropriately trained personnel and equipment for emergency resuscitation (including epinephrine and other emergency medicines) are readily available during infusion.

Who should not take Brineura?

  • Patients with any sign or symptom of acute or unresolved localized infection around the device insertion site (eg, cellulitis or abscess) or suspected or confirmed central nervous system (CNS) infection (eg, cloudy cerebrospinal fluid [CSF] or positive CSF gram stain, or meningitis)
  • Patients with active intraventricular access device-related complications (eg, leakage, device failure, or device-related infection, including meningitis)
  • Patients with shunts used to drain extra fluid around the brain

Administration: Brineura is only given by infusion into the fluid of the brain (known as an intraventricular injection) and using sterile technique to reduce the risk of infection. An intraventricular access device or port must be in place at least 5 to 7 days prior to the first infusion.

  • Prior to administration, it is important to discuss your child’s medical history with their doctor
  • Tell the doctor if your child is sick or taking any medication and if they are allergic to any medicines
  • Brineura is not recommended for use in patients less than 37 weeks post-menstrual age (gestational at birth plus post-natal age) or those weighing less than 2.5kg.

Meningitis and other device-related infections: Intraventricular access device-related infections, including meningitis, were observed with Brineura treatment. Infections required treatment with antibiotics and removal of the access device. If any signs of infection or meningitis occur, contact your child’s doctor immediately. The signs and symptoms of infections may not be readily apparent in patients with CLN2 disease.

  • Your child’s doctor should vigilantly be looking for signs and symptoms of infection, including meningitis, during treatment with Brineura
  • Your child’s doctor should inspect the scalp and collect samples of your child’s CSF prior to each infusion of Brineura, to check for infections and that there is no device failure
  • Signs of infection on or around the device insertion site may include redness, tenderness, or discharge

Device-related complications such as device leakage, device failure, leakage of CSF fluid, or bulging of the scalp around or above the intraventricular access device have occurred. In case of intraventricular access device-related complications, Brineura infusions may be discontinued.
Material degradation of the intraventricular access device reservoir was reported after approximately 4 years of administration, which may impact the effective and safe use of the device. During testing such material degradation was recognized after approximately 105 perforations of the intraventricular access device. The intraventricular access device should be replaced prior to 4 years of single-puncture administrations, which equates to approximately 105 administrations of Brineura.

Cardiovascular side effects: Low blood pressure and/or slow heart rate may occur during and following the infusion of Brineura. Contact your child’s healthcare provider immediately if these reactions occur. As part of the infusion, the healthcare provider will monitor vital signs (blood pressure, heart rate) before infusion starts, periodically during infusion, and post-infusion, and assess the patient’s status after administration to determine if continued observation may be necessary. Additional monitoring is required for patients with a history of cardiac abnormalities. In patients without cardiac abnormalities, regular 12-lead electrocardiogram (ECG) evaluations should be performed every 6 months.

Infusion Associated Reactions (IAR) such as vomiting, seizure, rash, pyrexia, hypersensitivity, and anaphylactic reaction have been observed in patients treated with Brineura. Your child’s doctor may prescribe medicines for your child to take 30 to 60 minutes prior to the start of infusion.

The most common side effects reported during Brineura infusions included:

  • Fever, problems with the electrical activity of the heart, decreased or increased protein in the fluid of the brain, vomiting, seizures, device-related complications, hypersensitivity, collection of blood outside of blood vessels (hematoma), headache, irritability, increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure.
  • The most frequent adverse reactions reported in patients less than 3 years of age treated with BRINEURA were similar to those observed in patients greater than 3 years of age except for hypersensitivity reactions, which were reported in 5 of 8 (63%) in patients less than 3 years of age at baseline compared with 0 of 6 in patients greater than 3 years of age at baseline. The most common manifestations of hypersensitivity were fever and vomiting. Such symptoms resolved over time or with administration of antipyretics, antihistamines and/or corticosteroids. Symptoms of severe hypersensitivity reactions (e.g., anaphylaxis) included rapid heartbeat, throat tightness, coughing, wheezing, trouble breathing, rash, diarrhea, hypotension, increased body temperature and vomiting.

The risk information provided here is not comprehensive. Talk to your healthcare provider to learn more or for medical advice about any side effects.

You may report side effects to BioMarin at 1-866-906-6100.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1–800-FDA-1088.

Please see accompanying full Prescribing Information with important warning for risk of anaphylaxis or visit www.Brineura.com.

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate BRINEURA in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue BRINEURA and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur

What is Brineura used for?

Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.