© 2021 BioMarin. All rights reserved.

The first approved treatment for any form of Batten disease.

Brineura® (cerliponase alfa) 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. Brineura is the first enzyme replacement therapy to be directly administered into the fluid of the brain, treating the underlying cause of CLN2 disease by helping to replace the deficient TPP1 enzyme missing in affected children.

Due to the potential for anaphylaxis, appropriate medical support should be readily available, and patients should be observed closely, during and after Brineura infusion. If anaphylaxis occurs, immediately discontinue infusion and initiate appropriate medical treatment. Inform patients/caregivers of the signs and symptoms of anaphylaxis and to seek immediate medical care should these occur. Consider the risks and benefits of readministration of Brineura following an anaphylactic reaction.

CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an estimated 20 children born in the United States each year–less than one in a million Americans.

Learn more about the development of the first therapy for CLN2 disease with “The Race for Brineura.”


Molecule
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)

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. The signs and symptoms of infections may not be readily apparent in patients with CLN2 disease. Your doctor should vigilantly be looking for signs and symptoms of infection, including meningitis, during treatment with Brineura.

Your child’s intraventricular access device should be replaced prior to 4 years of single-puncture administration of 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), symptom of acute, unresolved localized infection around the device insertion site (e.g. cellulitis or abscess), or and with shunts used to drain extra fluid around the brain. Your child’s doctor should inspect the scalp and collect samples of your child’s cerebrospinal fluid (CSF) prior to each infusion of Brineura, to check that there is no device failure or infections present.

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.


Image: Scientist handling beakers
Our Pipeline

We take on the biggest challenges in rare disease.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

Learn More about our pipeline