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BioMarin RareConnections: Uncommon Support for Rare Disease

Beyond the therapeutic support provided to children with CLN2 disease, BioMarin is committed to supporting healthcare professionals, family members, and caregivers.
BioMarin RareConnections provides personalized support services for caregivers at no cost, including education on CLN2 disease and Brineura, and information about financial assistance programs.
To enroll in BioMarin RareConnections, patients or their caregivers must sign and return the Patient Registration Forms. These forms can be obtained by contacting BioMarin RareConnections or by downloading them here >
Please contact BioMarin RareConnections if you would like assistance with either of these steps.

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Email: support@biomarin-rareconnections.com

BioMarin: A leader in rare disease

BioMarin has a 20-year history as a pioneer in developing therapies for rare diseases—no patient population is too small
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Resources for you and your patients

Download helpful materials, such as a Dosing and Administration Guide.

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). Consultation with a neurosurgeon may be needed to confirm the integrity of the 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 has occured after approximately 105 perforations of the intraventricular access device in benchtop testing, and has been observed in clinical trials at approximately 4 years of administration. Intraventricular access device replacement should be considered prior to 4 years of regular administration, equating 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, decreased CSF protein, vomiting, seizures, hypersensitivity, increased CSF protein, 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.
In clinical studies with Brineura, 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; antibiotics were administered, the intraventricular access device was replaced, and treatment continued. Device-related 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.
Intraventricular access device-related infections, including sub-clinical infections and meningitis, have been observed in patients treated with Brineura. Meningitis may present with the following symptoms: fever, headache, neck stiffness, light sensitivity, nausea, vomiting, and change in mental status. In clinical studies, antibiotics were administered, the intraventricular access device was replaced, and the patient continued on Brineura treatment.
Inform caregivers of the signs and symptoms of anaphylaxis, hypotension, bradycardia, and device-related complications and meningitis. 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 accompanying full Prescribing Information, or visit www.Brineura.com.
Indication
Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (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, including meningitis, were observed with Brineura treatment. If any signs of infection or meningitis occur, contact your child’s doctor immediately. Signs and symptoms of meningitis include but are not limited to fever, headache, neck stiffness, light sensitivity, nausea, vomiting and change in mental status.
Your child’s doctor may consider replacing your child’s intraventricular access device before the fourth year of treatment with Brineura, because the device may deteriorate due to repeated use.
Brineura should not be used in patients with active intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection, including meningitis) 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 BioMarin Pharmaceutical Inc. at 1-866-906-6100, or the FDA at 1-800-FDA-1088 or go to www.fda.gov/medwatch.
Please see accompanying full Prescribing Information, or visit www.Brineura.com.