{"id":26,"date":"2022-10-13T14:25:27","date_gmt":"2022-10-13T14:25:27","guid":{"rendered":"https:\/\/brineurahcp-dev-001.azurewebsites.net\/en-us\/brineura\/?page_id=26"},"modified":"2026-03-11T12:46:42","modified_gmt":"2026-03-11T12:46:42","slug":"about","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/brineura\/about\/","title":{"rendered":"About Brineura"},"content":{"rendered":"
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Brineura\u00ae (cerliponase alfa)\u2014the first and only treatment addressing the underlying cause of CLN2 disease1<\/sup><\/h1>\n

Brineura\u00ae (cerliponase alfa) injection for intraventricular infusion is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.1<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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CLN2 disease is a rare and rapidly progressing childhood neurodegenerative condition2<\/sup>\n<\/h3>\n

CLN2 disease is one of the most common forms of neuronal ceroid lipofuscinoses (NCLs),2<\/sup> also known as Batten disease. CLN2 disease is an autosomal recessive lysosomal storage disorder (LSD), and is associated with a predictable and rapid decline in function.2<\/sup><\/p>\n

Visit CLN2Connection.com<\/a> to learn more about CLN2 disease.<\/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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Brineura helps replace the deficient tripeptidyl peptidase 1 (TPP1) enzyme in children with CLN2 disease1<\/sup><\/h2>\n

Pathogenic variants (mutations) in the TPP1<\/em> gene (also referred to as the CLN2<\/em> gene) result in absence of or reduced activity of the TPP1 enzyme. This enzyme deficiency is associated with the neuronal degeneration observed in CLN2 disease.3<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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\n \"reduced <\/div>\n <\/figure>\n <\/div>\n
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Absence or reduced activity of the TPP1 enzyme is associated with an accumulation of storage material in the lysosome, which is associated with neuronal dysfunction and cell death.1,4<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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\n \"lysosomes\" <\/div>\n <\/figure>\n <\/div>\n
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Brineura is taken up into target cells and translocated to the lysosomes.1<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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\n \"lysosomal <\/div>\n <\/figure>\n <\/div>\n
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Brineura cleaves the lysosomal tripeptides and helps to clear accumulation in the cell.1<\/sup><\/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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Brineura is contraindicated in patients with any sign or symptom of acute or unresolved localized infection on or around the device insertion site, suspected or confirmed CNS infection, acute intraventricular access device-related complications, and ventriculoperitoneal shunts. Hypersensitivity reactions, intravascular access device-related infections and complications, cardiovascular reactions, and infusion-associated reactions related to Brineura may occur.1<\/sup><\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\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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Brineura is an enzyme replacement therapy administered via intraventricular infusion1<\/sup><\/h3>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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The use of intraventricular administration ensures Brineura is delivered directly into the central nervous system. Intraventricular delivery has been used in clinical settings for a long period of time in adults and children, and is an established approach for delivery of drugs to the brain.5<\/sup><\/p>\n

Brineura is administered to the cerebrospinal fluid by infusion via a surgically implanted reservoir and catheter (intraventricular access device). The intraventricular access device must be surgically implanted prior to the first intraventricular infusion. It is recommended that the first dose be administered at least 5 to 7 days after device implantation.1<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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Brineura is contraindicated if there are signs of acute intraventricular access device-related complications. In case of intraventricular access device complications, discontinue the Brineura infusion and refer to the manufacturer’s labeling for further instructions.1<\/sup><\/strong><\/p>\n

Device-related adverse reactions were reported in 12 of 24 patients in Trial 1 & 2 and 3 of 14 patients in Trial 3. Adverse reactions included infection, delivery system-related complications, pleocytosis, device breakage, device leakage and CSF red blood cell count positive.1<\/sup><\/strong><\/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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Initiate Brineura in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.1<\/sup><\/strong><\/h3>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Brineura Dosage<\/h2>\n <\/div>\n\n
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Brineura Dose, Volume, and Infusion Rate by Age1<\/sup><\/h2>\n <\/div>\n\n
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The dose is administered once every other week by intraventricular infusion.<\/p>\n

Brineura is not recommended in patients less than 37 weeks post-menstrual age (gestational age at birth plus post-natal age) or those weighing less than 2.5 kg.<\/strong><\/p>\n

Administration of Brineura should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis.<\/p>\n

Administer Brineura first followed by infusion of the Intraventricular Electrolytes. The complete Brineura infusion, including the required infusion of Intraventricular Electrolytes, is approximately 2 to 4.5 hours, depending on the dose and volume administered.<\/p>\n

If one or more doses are missed, restart Brineura treatment as soon as possible maintaining the 2-week interval between infusions thereafter.<\/p>\n

Brineura should be administered, under strict aseptic techniques. Administration protocols may vary by institution. Refer to your institution\u2019s policies and procedures for guidance.<\/p>\n

The care team prepares the patient and their family for infusion and what to expect during the infusion process. The child will be monitored before, during, and after the infusion. Pretreatment of patients with antihistamines with or without antipyretics or corticosteroids is recommended 30 to 60 minutes prior to the start of the infusion.<\/p>\n

Perform electrocardiogram (ECG) monitoring during infusion in patients with a history of bradycardia, conduction disorder, or with structural heart disease, as some patients with CLN2 disease may develop conduction disorders or heart disease. In patients without cardiac abnormalities, regular 12-lead ECG evaluations should be performed every 6 months.<\/p>\n

Learn more about Brineura administration by downloading a Dosing and Administration Guide ><\/span><\/a>
\nView more helpful resources ><\/span><\/a><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Connect with BioMarin to register for Brineura updates\n<\/h4>\n \n \n <\/div>\n
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Register Now<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Brineura safety was evaluated in 38 patients with CLN2 disease across multiple clinical studies\n<\/h4>\n \n \n <\/div>\n
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See Safety Profile<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Download helpful materials for you and your patients\n<\/h4>\n \n \n <\/div>\n
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View Resources<\/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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See Brineura Efficacy Results\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

The efficacy of Brineura was assessed in multiple clinical trials including in children below 3 years of age.<\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

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Next Section<\/a><\/p>\n <\/div>\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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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 S, Williams R, Goebel HH. Neuronal ceroid-lipofuscinoses. In: Pagon RA, Adam MP, Ardinger HH, et al, eds. GeneReviews<\/em>\u00ae<\/sup> [Internet]. Seattle, WA: University of Washington, Seattle; 1993\u20132016. Published October 10, 2001.\n<\/span><\/li>\n
  3. Schulz A, Kohlsch\u00fctter A, Mink J, Simonati A, Williams R. NCL diseases \u2013 clinical perspectives. Biochim Biophys Acta<\/em>. 2013;1832:1801-1806.\n<\/span><\/li>\n
  4. Vuillemenot BR, Kennedy D, Cooper JD, et al. Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis. Mol Genet Metab<\/em>. 2015;114:281-293.\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;67:23-25.\n<\/span><\/li>\n <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":4,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-26","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nAbout Brineura - BioMarin Brineura HCP EN-US<\/title>\n<meta name=\"description\" content=\"Brineura\u00ae (cerliponase alfa) injection for intraventricular infusion is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/hcp.biomarin.com\/en-us\/brineura\/about\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"About Brineura\" \/>\n<meta property=\"og:description\" content=\"Brineura\u00ae (cerliponase alfa) injection for intraventricular infusion is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency\" \/>\n<meta property=\"og:url\" content=\"https:\/\/hcp.biomarin.com\/en-us\/brineura\/about\/\" \/>\n<meta property=\"og:site_name\" content=\"BioMarin Brineura HCP EN-US\" \/>\n<meta property=\"article:modified_time\" content=\"2026-03-11T12:46:42+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/hcp.biomarin.com\/en-us\/brineura\/wp-content\/uploads\/sites\/2\/2026\/01\/cropped-brin-favicon-1-e1706638112801.png?v=0.31\" \/>\n\t<meta property=\"og:image:width\" content=\"512\" \/>\n\t<meta property=\"og:image:height\" content=\"512\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/png\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/brineura\\\/about\\\/\",\"url\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/brineura\\\/about\\\/\",\"name\":\"About Brineura - 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