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Brineura(cerliponase alfa) for CLN2 Disease

Brineura (cerliponase alfa) is the first approved treatment for any form of Batten disease, and 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.i CLN2 disease is an ultra-rare and rapidly progressing brain disorder that affects an approximately estimated 20 children born in the United States each year– less than one in a million Americans.2,3

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.4

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, and undesirable hypersensitivity reactions, including fever, vomiting, irritability, and anaphylaxis, may occur during and following the Brineura infusion. Please see important safety information, below.

Brineura At-a-Glance5

  • First approved therapy for any form of Batten disease
  • Designated as an orphan drug in the United States and the European Union
  • Manufactured and commercialized by BioMarin

Regulatory Status

In April 2017, Brineura was approved by the U.S. Food and Drug Administration. Also in April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion for the company’s Marketing Authorization Application for Brineura. The CHMP’s recommendation was referred to the European Commission (EC), which is expected to render a final decision in the third quarter of 2017. If approved by the EC, BioMarin will receive marketing authorization for Brineura in all European Union member states.

Commercialization Plan

Brineura is manufactured and commercialized by BioMarin in the United States.

Additional Resources

For additional information, please visit the following websites and others listed in the Patient/Physician Resource Library:

Please see Important Safety Information below

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 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 accompanying full Prescribing Information, or visit www.Brineura.com.


1 Schulz A, Kohlschütter A, Mink J, Simonati A, Williams R. NCL diseases– clinical perspectives. Biochim Biophys Acta. 2013;1832:1801-1806.
2 Claussen M, Heim P, Knispel J, Goebel HH, Kohlschütter A. Incidence of neuronal ceroid-lipofuscinoses in West Germany: variation of a method for studying autosomal recessive disorders. Am J Med Genet. 1992;42:536-538
3 Martin J. A., Hamilton B. E., Ventura S. J., Osterman, M. J., Wilson, E. C., Mathews, T. J., et al. (2011). Births: Final data for 2010. National Vital Statistics Reports, 61, 1–72. Retrieved January 21, 2013, from http://www.cdc.gov/nchs/data/nvsr/nvsr61/nvsr61_01.pdf#table01 (PDF – 1.66 MB)
4 Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; 2017.
5 BioMarin. BioMarin Doses First Patient in Phase 1/2 Trial With BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease [Press Release]. 2013. Retrieved from:
http://investors.biomarin.com/2013-09-23-BioMarin-Does-First-Patient-in-Phase-1-2-Trial-With-BMN-190-for-the-Treatment-of-Neuronal-Ceroid-Lipofuscinosis-Type-2-a-Form-of-Batten-Disease

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Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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