{"id":37,"date":"2024-11-20T14:52:19","date_gmt":"2024-11-20T14:52:19","guid":{"rendered":"https:\/\/www.brineura.com\/en-us\/?page_id=37"},"modified":"2026-03-12T13:43:34","modified_gmt":"2026-03-12T13:43:34","slug":"about-brineura","status":"publish","type":"page","link":"https:\/\/www.brineura.com\/en-us\/about-brineura\/","title":{"rendered":"About Brineura"},"content":{"rendered":"
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Brineura® (cerliponase alfa) is the only treatment that directly addresses the cause of CLN2 disease by helping to replace the TPP1 enzyme1<\/sup><\/h1>\n <\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n
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What is Brineura?<\/h2>\n

Brineura® (cerliponase alfa<\/span>) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.1<\/sup><\/p>\n

CLN2 disease<\/span> is a type of lysosomal storage disorder that affects cells in the brain. Lysosomes are in every cell and contain enzymes that break down materials in the cell. One of these enzymes is called TPP1, which is missing or not working properly in children with CLN2 disease.2<\/sup><\/p>\n <\/div>\n\n

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The TPP1 enzyme<\/span><\/span> is missing or not working properly in children with CLN2 disease<\/span>. This leads to a buildup of storage materials in their lysosomes<\/span>, associated with cell damage in the brain.2-4<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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When Brineura is delivered to a child with CLN2 disease, it helps replace the missing or malfunctioning TPP1 enzyme.1<\/sup><\/p>\n

Learn more about CLN2 disease<\/a><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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How is Brineura administered to my child?<\/h2>\n

Brineura is a type of treatment called enzyme replacement therapy (ERT)<\/span><\/span>. It’s administered through intraventricular infusion<\/span><\/span>—a method that allows Brineura to be directly delivered into the fluid surrounding the brain, known as the cerebrospinal fluid<\/span>.1<\/sup><\/p>\n

Before starting Brineura, your child will need to have an intraventricular access device<\/span><\/span> surgically implanted just below their scalp. This is an established procedure in pediatric neurology, and allows direct delivery of Brineura into a ventricle<\/span><\/span> in the brain. The access device is about the size of a penny. It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.1,5<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Brineura treatment will take approximately 2 to 4.5 hours, depending on the dose and volume administered, every other week. You’ll work with your healthcare team to schedule treatments.1<\/sup><\/p>\n

Your healthcare team will let you know how to prepare your child for infusion<\/span><\/span>, and what to expect during this procedure. Your child will be monitored before, during, and after the infusion<\/span>, and may receive medications to reduce the risk of hypersensitivity<\/span><\/span> reactions.1<\/sup><\/p>\n

Your doctor may also take the steps to minimize the risk of device-related infections at every treatment. You may be asked to wear a mask and the number of people in the room will be limited. Ask your team who and what is allowed in the room.1<\/sup><\/p>\n

If you see signs of infection, such as swelling or reddening of the skin, contact your healthcare team immediately.1<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Matty’s infusion day<\/h2>\n

Matty is a patient with CLN2 disease<\/span>. He’s been on treatment with Brineura since 2015.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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After the first few infusions we became more comfortable. The initial surgery for the port was scary, so we felt like that was the first hurdle to jump. Once Matty had a few infusions, we became more comfortable with the process.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t —Joe, Matty’s dad<\/span><\/cite>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/blockquote>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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Connect with BioMarin to register for Brineura updates<\/h5>\n \n <\/div>\n
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Register now<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Discover the path toward treatment with Brineura<\/h5>\n \n <\/div>\n
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Getting Started<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Learn how Brineura helps children with CLN2 disease<\/h5>\n \n <\/div>\n
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Treatment<\/a><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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References:\n<\/h4>\n\t\t\t\t\t\t
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  1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n
  2. Mole SE, Williams RE, Goebel HH. Correlations between genotype, ultrastructural morphology and clinical phenotype in the neuronal ceroid lipofuscinoses. Neurogenetics<\/em>. 2005;6:107-126.\n<\/span><\/li>\n
  3. Haltia M. The neuronal ceroid-lipofuscinoses: from past to present. Biochim Biophys Acta<\/em>. 2006;1762:850-856.\n<\/span><\/li>\n
  4. Schulz A, Kohlschütter A, Mink J, Simonati A, Williams R. NCL diseases – clinical perspectives. Biochim Biophys Acta<\/em>. 2013;1832:1801-1806.\n<\/span><\/li>\n
  5. Cohen-Pfeffer JL, Gururangan S, Lester T, et al. Intracerebroventricular delivery as a safe, long-term route of drug administration. Pediatr Neurol<\/em>. 2017;67:23-35.\n<\/span><\/li>\n <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n

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