Resources for you and your family
Connect with a growing community of support for CLN2 disease
Caring for a child who has CLN2 disease can be emotionally and physically demanding. Connecting with other parents and healthcare professionals can help make the disease easier to manage. Fortunately, there are support resources available to you and your family.

Batten Disease Support and Research Association (BDSRA)
The BDSRA is committed to providing family support services and has experience coordinating travel logistics for CLN2 families. If you have a child with CLN2 disease, the BDSRA welcomes you and wants to answer your questions.
Join the BDSRA community at www.bdsra.org or 1-800-488-4570. Contact Tracy Kirby, BDSRA’s Family Liaison at tkirby@bdsra.org.

Connect at BDSRA.org

Noah’s Hope
Created by a family with 2 children affected by CLN2 disease, this organization has raised funds for research all over the world.

Connect at NoahsHope.com

Hope4Bridget
Dedicated to raising awareness and funding for research, this site shares support and experience from a family raising a daughter with CLN2 disease.

Connect at Hope4Bridget.com
CLN2 disease website
CLN2 Family is an educational resource about CLN2 disease for you and your family.

Visit CLN2Family.com
Layla and family
Layla is a patient with CLN2 disease.
She’s been on treatment with Brineura since 2015.

“Newly diagnosed parents should reach out to the BDSRA. They are there for just that, support. They can connect you to other families going through similar stories. They can just point you in the right direction. They’re the best people to reach out to.”

—Maria, Layla’s mom
Resources for you and your loved ones
Download the following materials to help guide you on your own Brineura journey.

BioMarin RareConnections™ patient registration forms
Complete and return these forms to enroll your child in BioMarin RareConnections™. This program provides individual support for patients and their families throughout the Brineura treatment process.

Download >
Beginning with Brineura
This convenient brochure gives you a quick introduction to Brineura® (cerliponase alfa) as you get started with treatment.

Download >
Preparing for Brineura infusions
This useful brochure helps you and your family prepare for what to expect before, during, and after infusions.

Download >
Roadmap to Brineura
This roadmap highlights major steps that you and your child may take on your road to Brineura treatment.

Download >
Glossary of terms
This glossary includes common words or phrases associated with CLN2 disease that may be new to you.

Download >
Brineura Prescribing Information
This document educates patients about Brineura. It includes instructions for use and safety information.

Download >
Learn more about Brineura and how it’s administered
Brineura is the only treatment that directly addresses the cause of CLN2 disease.

Resources for you and your family

Connect with a growing community of support for CLN2 disease

Caring for a child who has CLN2 disease can be emotionally and physically demanding. Connecting with other parents and healthcare professionals can help make the disease easier to manage. Fortunately, there are support resources available to you and your family.

Batten Disease Support and Research Association (BDSRA)

The BDSRA is committed to providing family support services and has experience coordinating travel logistics for CLN2 families. If you have a child with CLN2 disease, the BDSRA welcomes you and wants to answer your questions.
Join the BDSRA community at www.bdsra.org or 1-800-488-4570. Contact Morgan DeBoth, BDSRA’s Manager of Family Support at mdeboth@bdsra.org.

Noah’s Hope

Created by a family with 2 children affected by CLN2 disease, this organization has raised funds for research all over the world.

Hope4Bridget

Dedicated to raising awareness and funding for research, this site shares support and experience from a family raising a daughter with CLN2 disease.

cln2family_website_844_border
CLN2 disease website

CLN2 Family is an educational resource about CLN2 disease for you and your family.

layla_and_family_original

Layla and family

Layla is a patient with CLN2 disease.
She's been on treatment with Brineura since 2015.

“Newly diagnosed parents should reach out to the BDSRA. They are there for just that, support. They can connect you to other families going through similar stories. They can just point you in the right direction. They're the best people to reach out to.”

—Maria, Layla’s mom

Resources for you and your loved ones

Download the following materials to help guide you on your own Brineura journey.

patient_registration_forms_844_border
BioMarin RareConnections™ patient registration forms

Complete and return these forms to enroll your child in BioMarin RareConnections™. This program provides individual support for patients and their families throughout the Brineura treatment process.

beginning_with_brineura_844_border
Starting Brineura

This convenient brochure gives you a quick introduction to Brineura® (cerliponase alfa) and helps you and your family prepare for treatment.

preparing_for_brineura_infusions_844_border
Preparing for Brineura infusions

This useful brochure helps you and your family prepare for what to expect before, during, and after infusions.

roadmap-to-brin-2
Roadmap to Brineura

This roadmap highlights major steps that you and your child may take on your road to Brineura treatment.

glossary_844_border
Glossary of terms

This glossary includes common words or phrases associated with CLN2 disease that may be new to you.

prescribing_information_844_border
Brineura Prescribing Information

This document educates patients about Brineura. It includes instructions for use and safety information.

Learn more about Brineura and how it’s administered

Brineura is the only treatment that directly addresses the cause of CLN2 disease.

US-BRIN-00058 0623

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
Contraindications

Brineura is contraindicated in patients with:

  • any sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g., cellulitis or abscess); or suspected or confirmed CNS infection (e.g., cloudy CSF or positive CSF gram stain, or meningitis)
  • any acute intraventricular access device-related complications (e.g., leakage, extravasation of fluid, or device failure)
  • ventriculoperitoneal shunts
Important Preparation and Administration Information
Brineura must only be administered via the intraventricular route using aseptic technique to reduce the risk of infection. Administer Brineura and the Intraventricular Electrolytes using the provided Administration Kit for use with Brineura components. Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage or failure and for potential infection. Prior to each infusion of Brineura and when clinically indicated, send cerebrospinal fluid (CSF) samples for testing of cell count and culture.
Special Populations

Brineura has not been studied in pregnancy or lactation.

Safety and effectiveness in pediatric patients below 3 years of age have not been established.

WARNINGS AND PRECAUTIONS
Meningitis and Other Intraventricular Access Device-Related Infections
Bacterial meningitis requiring antibiotic treatment and removal of the device was reported during postmarketing use of Brineura. The signs and symptoms of infections may not be readily apparent in patients with CLN2 disease. To reduce the risk of infectious complications, Brineura should be administered by, or under the direction of, a physician experienced in intraventricular administration.
Intraventricular Access Device-Related Complications
During the clinical trial and in postmarketing reports, intraventricular access device-related complications were reported (e.g., device leakage, device failure, extravasation of CSF fluid, or bulging of the scalp around or above the intraventricular access device). In case of intraventricular access device-related complications, discontinue the Brineura infusion and refer to the device manufacturer’s labeling for further instructions.
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. The intraventricular access device should be replaced prior to 4 years of single-puncture administrations, which equates to approximately 105 administrations of Brineura.
Cardiovascular Adverse Reactions
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.
Hypersensitivity Reactions, Including Anaphylaxis
Hypersensitivity reactions, including anaphylaxis, have been reported in Brineura-treated patients during clinical studies and postmarketing use. In clinical trials, a total of 11 out of 24 patients (46%) experienced hypersensitivity reactions during the infusion or within 24 hours of completion of the infusion.
Due to the potential for anaphylaxis, appropriate medical support should be readily available when Brineura is administered. If a severe hypersensitivity reaction or anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Observe patients closely during and after the infusion. Inform patients/caregivers of the signs and symptoms of hypersensitivity reactions and anaphylaxis and instruct them to seek immediate medical care should signs and symptoms occur. Consider the risks and benefits of readministration of Brineura following an anaphylactic reaction.
ADVERSE REACTIONS
In clinical trials, the most frequently reported adverse reactions (≥8%) were pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, device-related complications, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.
Seizures were reported in 12 of 24 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.
Adverse reactions related to the device were observed in 12 of 24 patients. Device-related adverse reactions include infection, delivery system-related complications, and 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%) in patients treated with Brineura. No association was found between serum or CSF ADA titers and incidence or severity of hypersensitivity.
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.
What is Brineura?
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.
Who should not take Brineura?
  • Patients with active intraventricular access device-related complications (eg, leakage, device failure, or device-related infection, including meningitis)
  • 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 shunts used to drain extra fluid around the brain

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

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 they are sick or taking any medication and if they are allergic to any medicines

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, extravasation 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.
Hypersensitivity reactions including serious and severe allergic reactions (anaphylaxis) may occur. Symptoms of anaphylaxis may include fever, respiratory distress, rash, vomiting, and irritability, and may occur during treatment or within several hours of Brineura infusion. Seek immediate medical care should signs and symptoms of anaphylaxis occur. Your child may receive medication such as antihistamines before Brineura infusions to reduce the risk of reactions.
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 and will start the subsequent infusion at approximately one-half the initial infusion rate at which the anaphylactic reaction occurred.

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 risk information provided here is not comprehensive. Talk to your healthcare provider to learn more or for medical advice about any side effects.
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 click here to see full Prescribing Information or visit www.Brineura.com.