Discover the path toward treatment with Brineura® (cerliponase alfa)

Every child’s journey with Brineura® (cerliponase alfa), an enzyme replacement therapy, will be different. Learn about how to get started.

Your doctor will determine if
Brineura is right for your child

You'll consult with your child’s doctor and, together, determine whether Brineura is right for your child.

Your child's doctor may consult with other experts more familiar with CLN2 disease. You and your child may need to travel to one of these experts at a different hospital to receive treatment.

DOCTOR’S APPOINTMENT

You'll consult with your child’s doctor and, together, determine whether Brineura is right for your child.

Your child’s doctor may consult with other experts more familiar with CLN2 disease. You and your child may need to travel to one of these experts at a different hospital to receive treatment.

Roadmap to Brineura

Your healthcare team at the hospital will work on an individualized plan for your child, which your doctor will discuss with you. This roadmap is meant to give you an overview of the path toward treatment. Your family may have a similar path, or your path may vary.

Enroll your child with BioMarin RareConnections

Individualized support available through BioMarin RareConnections

Beyond the therapeutic support provided to children with CLN2 disease, BioMarin is committed to supporting family members and caregivers. BioMarin RareConnections™ provides a variety of personalized support services at no cost, including education on CLN2 disease and Brineura, and support to coordinate additional services, such as information about financial assistance programs.

Contact the BioMarin RareConnections™ team for more information by emailing support@biomarin-rareconnections.com or by calling 1-866-906-6100.

Complete and return the Patient Registration Forms to enroll your child.

Your doctor will create a treatment plan specific to your child’s needs

HOSPITAL TREATMENT PLAN

Brineura is a unique therapy, so creating a treatment plan specific to your child’s needs may take some time.

Brineura requires a multidisciplinary team—that means many people from different departments in the hospital will be involved. The hospital will work with your insurance provider to establish reimbursement for Brineura therapy.

As a caregiver, you’re the most important part of your child’s team. You are your child's advocate and a key source of information, whether it’s medical records or insights into your child's well-being.

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

On infusion days: “Matty loves his iPad and enjoys playing games, looking at books, and watching movies on it. The iPad makes life so much easier for him (and us) in the hospital.”

—Joe, Matty's dad

Before starting Brineura

Brineura is administered every other week through intraventricular infusion.1This method allows Brineura to be delivered directly to a ventricle in the brain, and then into the fluid surrounding the brain, known as the cerebrospinal fluid (CSF). Brineura is delivered into the CSF to help reach cells that are affected by CLN2 disease. Knowledgeable members of your healthcare team will give your child’s Brineura infusions.

To receive intraventricular infusions, your child will first need to have an intraventricular access device surgically implanted.1 This is an established procedure in pediatric neurosurgery.2 The neurosurgeon will discuss the procedure with you and answer any additional questions you may have.

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 infection.

Low blood pressure and/or slow heart rate, and undesirable hypersensitivity reactions, including fever, vomiting, irritability, and anaphylaxis, may occur during and following the Brineura infusion.

After your child has been prescribed Brineura, some of the steps may include:

MRI BRAIN SCANS3

MRI scans are used to help the surgeon locate where the intraventricular access device should be inserted and to confirm placement after surgery.

THE INTRAVENTRICULAR ACCESS DEVICE WILL BE SURGICALLY IMPLANTED

This established procedure is necessary for your child to receive Brineura.1,2 It allows direct delivery of Brineura into a ventricle in the brain.

BRINEURA INFUSIONS CAN BEGIN

It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.1 Work with your healthcare team to schedule treatments.

Brineura treatment will take about 4.5 hours every other week1

MRI, magnetic resonance imaging.

Connect with BioMarin

Learn more about Brineura by registering to receive updates.

References: 1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2017. 2. Data on file, BioMarin Pharmaceutical Inc. 3. Steinfeld R, Heim P, von Gregory H, et al. Late infantile neuronal ceroid lipofuscinosis: quantitative description of the clinical course in patients with CLN2 mutations. Am J Med Genet. 2002;112:347-354. 4. Nickel M, Jacoby D, Lezius S, et al. Natural history of CLN2 disease: quantitative assessment of disease characteristics and rate of progression. Poster session presented at: The 12th Annual WORLD Symposium; February – March 2016; San Diego, CA. 5. Fietz M, AlSayed M, Burke D, et al. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis. Mol Genet Metab. 2016;119:160-167.

Indication
Brineura® (cerliponase alfa) injection for intraventricular use is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Important Safety Information
Brineura is contraindicated in patients with acute intraventricular access device-related complications and with ventriculoperitoneal shunts.
Brineura must only be administered via the intraventricular route and using aseptic technique to reduce the risk of infection. Healthcare professionals should inspect the scalp for skin integrity to ensure the intraventricular access device is not compromised prior to each infusion. Brineura is contraindicated if there are signs of acute intraventricular access device-related complications (e.g., leakage, device failure or signs of device-related infection such as swelling, erythema of the scalp, extravasation of fluid, or bulging of the scalp around or above the intraventricular access device). In case of intraventricular access device complications, discontinue the Brineura infusion and refer to the manufacturer’s labeling for further instructions. Routinely send cerebrospinal fluid (CSF) samples for testing to detect subclinical device infections.
Material degradation of the intraventricular access device reservoir may occur after approximately 105 perforations of the intraventricular access device and may require replacement as soon as, or prior to, 105 administrations of Brineura.
Monitor vital signs before infusion starts, periodically during infusion, and post-infusion in a healthcare setting. Perform electrocardiogram (ECG) monitoring during infusion in patients with a history of bradycardia, conduction disorder, or with structural heart disease. In patients without cardiac abnormalities, regular 12-lead ECG evaluations should be performed every 6 months.
Hypotension occurred in 2 patients during or up to 8 hours after Brineura infusion. Patients did not require alteration in treatment, and reactions resolved spontaneously or after intravenous fluid administration.
One patient experienced hypoxia 8 hours after Brineura infusion, followed by a low mean arterial pressure at 15 hours post infusion. Symptoms resolved after oxygen administration, airway repositioning, and normal saline infusion. One patient reported decreased oxygen saturation, 45 minutes after starting Brineura, with associated low diastolic blood pressure. Hypoxia resolved after oxygen administration. No treatment was administered for the low diastolic blood pressure, which returned to normal while the patient continued to receive Brineura infusion without change to the infusion rate or dose.
Due to the potential for anaphylaxis, appropriate medical support should be readily available when Brineura is administered. If anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Observe patients closely during and after the infusion.
Hypersensitivity reactions were reported in 11 patients during or within 24 hours after completion of the Brineura infusion. The signs and symptoms observed concomitantly with hypersensitivity reactions include pyrexia, vomiting, pleocytosis, or irritability. Patients were routinely premedicated with antihistamines with or without antipyretics or corticosteroids, prior to infusion of Brineura.
The management of hypersensitivity reactions should be based on the severity of the reaction and may include temporarily interrupting the infusion, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If a severe hypersensitivity reaction occurs, immediately discontinue the infusion and initiate appropriate medical treatment.
Brineura has not been studied in pregnancy or lactation.
Safety and effectiveness in pediatric patients below 3 years of age have not been established.
In clinical trials, the most frequently reported adverse reactions (≥8%) were pyrexia, ECG abnormalities, CSF protein decreased, vomiting, seizures, hypersensitivity, CSF protein increased, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.
Seizures were reported in 12 patients and included atonic, generalized tonic-clonic, focal, and absence. Seizures were managed with standard anticonvulsive therapies and did not result in discontinuation of Brineura treatment.
Device-related adverse reactions were reported in 12 patients and included infection, delivery system–related complications, and pleocytosis. Intraventricular access device-related CNS infections were observed in 2 patients. In both cases, antibiotics were administered, the intraventricular access device was replaced, and treatment continued. Devicerelated complications did not result in discontinuation of Brineura treatment. Other device-related adverse reactions included 1 patient with leakage of the intraventricular access device and 1 with pleocytosis.
Hematoma adverse reactions were reported in 5 patients and presented as hematoma, post procedural hematoma, traumatic hematoma, and subdural hematoma. Hematomas did not require treatment and did not interfere with Brineura infusion.
Anti-drug antibodies (ADAs) were detected in serum (79%) and CSF (33.3%) in patients treated with Brineura. No association was found between serum or CSF ADA titers and incidence or severity of hypersensitivity.
Inform caregivers of the signs and symptoms of anaphylaxis, hypotension, bradycardia, and device-related complications. Instruct them to seek immediate medical care should any of these signs and symptoms occur.
To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088, or go to www.fda.gov/medwatch.
Please see full Prescribing Information
Indication
Brineura® (cerliponase alfa) injection for intraventricular use is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Important Safety Information
Brineura is a prescription medicine. Before treatment with Brineura, it is important to discuss your child’s medical history with their doctor. Tell the doctor if they are sick or taking any medication and if they are allergic to any medicines. Your child’s doctor will decide if Brineura is right for them. If you have questions or would like more information about Brineura, contact your child’s doctor.
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. Intraventricular access device-related infections were observed with Brineura treatment. If any signs of infection occur, contact your child’s doctor immediately. Your child’s intraventricular access device may need to be replaced over time.
Brineura should not be used in patients with active intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection) and with shunts used to drain extra fluid around the brain.
Low blood pressure and/or slow heart rate may occur during and following the Brineura infusion. Contact your child’s doctor immediately if these reactions occur.
Undesirable or hypersensitivity reactions related to Brineura treatment, including fever, vomiting, and irritability, may occur during treatment and as late as 24 hours after infusion. Your child may receive medication such as antihistamines before Brineura infusions to reduce the risk of reactions. Serious and severe allergic reactions (anaphylaxis) may occur. If a reaction occurs, the infusion will be stopped and your child may be given additional medication. If a severe reaction occurs, the infusion will be stopped and your child will receive appropriate medical treatment. If any signs of anaphylaxis occur, immediately seek medical care.
Safety and effectiveness in pediatric patients below 3 years of age have not been established.
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, hypersensitivity, collection of blood outside of blood vessels (hematoma), headache, irritability, and increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure. Intraventricular device-related side effects included infection, delivery system-related complications, and increased white blood cell count in fluid of the brain.
These are not all of the possible side effects with Brineura. Talk to your child’s doctor if they have any symptoms that bother them or that do not go away.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see full Prescribing Information