{"id":166,"date":"2022-10-14T16:29:35","date_gmt":"2022-10-14T16:29:35","guid":{"rendered":"https:\/\/brineurahcp-dev-001.azurewebsites.net\/en-us\/brineura\/?page_id=166"},"modified":"2026-03-11T12:51:08","modified_gmt":"2026-03-11T12:51:08","slug":"safety","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/brineura\/safety\/","title":{"rendered":"Safety Profile"},"content":{"rendered":"
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Brineura\u00ae (cerliponase alfa) safety profile<\/h1>\n

Adverse reactions reported in \u2265 <\/strong>8% of symptomatic pediatric patients with CLN2 disease in Brineura Trial 1 and Trial 2 at week 961<\/sup><\/h2>\n

Trials 1 and 21<\/sup><\/h3>\n

The safety of Brineura\u00ae<\/sup> (cerliponase alfa) was evaluated in 24 patients with CLN2 disease who received at least 1 dose of Brineura in a clinical study with extension of up to 161 weeks.1<\/sup><\/p>\n <\/div>\n\n

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*Increased body temperature includes: increased body temperature and pyrexia.<\/small>
\n\u2020<\/sup>ECG abnormalities include: non-specific repolarization abnormality, notched QRS, ST segment elevation, biphasic T wave abnormality, supraventricular extrasystoles,bradycardia, sinus tachycardia, and intraventricular conduction delay.<\/small>
\n\u2021<\/sup>Seizures include: atonic, generalized tonic-clonic, focal, and absence.<\/small>
\n\u00a7<\/sup>Device-related complications include device-related infection, delivery system-related complications (needle issues, device leakage, device malfunction, device difficult to use, medical device site irritation, device breakage, etc) and pleocytosis.<\/small>
\n||<\/sup>Hypersensitivity includes only hypersensitivity.<\/small>
\n\u00b6<\/sup>Device-related infections include: Propionibacterium acnes<\/i> and Staphylococcus epidermidis<\/i>.<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Trial 3<\/h3>\n

The most frequent adverse reactions reported in patients <3 years treated with Brineura were similar to those observed in patients \u22653 years of age except for hypersensitivity reactions, which were reported in 5 of 8 (63%) patients <3 years at baseline compared with 0 of 6 in patients \u22653 years of age at baseline. The most common manifestations of hypersensitivity were pyrexia and vomiting and the timing and resolution were similar to Trials 1 and 2.1<\/sup><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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ADVERSE REACTIONS REPORTED IN \u22655% OF PEDIATRIC PATIENTS\u00a0<\/sup>(1 TO 6 YEARS OF AGE) WITH CLN2 DISEASE TREATED WITH BRINEURA IN TRIAL 31<\/sup><\/h3>\n <\/div>\n\n
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Note: Incidence numbers are based on baseline age group.<\/p>\n

*ECG abnormalities include: non-specific repolarization abnormality, supraventricular extrasystoles, possible left ventricular hypertrophy, intermittent 2nd degree AV Block (type 2 Mobitz), incomplete right bundle branch block, and prominent Q wave.
\n\u2020<\/sup>Increased body temperature includes: increased body temperature and pyrexia.
\n\u2021<\/sup>Seizures include: atonic, febrile convulsion, generalized tonic-clonic, partial, partial with secondary generalization, petit mal epilepsy, myoclonic and status epilepticus.
\n\u00a7<\/sup>Device-related complications include device-related infection, delivery system-related complications (needle issues, device leakage, device malfunction, device difficult to use, medical device site irritation, device breakage, etc).
\n||<\/sup>Hypersensitivity includes only the term of hypersensitivity.
\n\u2022 In Trial 3, infusion-associated reactions were reported in 8 of 8 patients less than 3 years of age at baseline as compared with 4 of 6 patients \u22653 years of age at baseline<\/p>\n

ECG, electrocardiogram.<\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

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Because clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical studies of a drug cannot be directly compared to rates in the clinical studies of another drug and may not reflect the rates observed in practice.<\/p>\n

Due to the potential for anaphylaxis, appropriate medical support should be readily available including access to cardiopulmonary resuscitation equipment, 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.<\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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To report suspected adverse reactions, contact:
\nBioMarin Pharmaceutical Inc.<\/strong>
\nPhone: 1-866-906-6100
\nEmail: drugsafety@bmrn.com<\/a><\/p>\n

FDA<\/strong>
\nPhone: 1-800-FDA-1088
\nWeb: www.fda.gov\/medwatch<\/a><\/p>\n <\/div>\n \n

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For medical information inquiries:
\nBioMarin\u00a0Medical department<\/strong>
\nEmail: medinfo@bmrn.com<\/a>
\nPhone: 1-800-983-4587<\/p>\n <\/div>\n \t\t<\/div>\n\t<\/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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The efficacy of Brineura was assessed in multiple clinical trials including in children below 3 years of age\n<\/h4>\n \n \n <\/div>\n
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See Efficacy Results<\/a><\/p>\n <\/div>\n<\/div>\n\n

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Brineura is an enzyme replacement therapy that helps replace deficient TPP1 enzyme\n<\/h4>\n \n \n <\/div>\n
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Learn how Brineura Works<\/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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Connect With BioMarin\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Register to get Brineura updates, as well as helpful resources and information.<\/strong><\/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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Reference:\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 <\/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-166","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nSafety Profile - BioMarin Brineura HCP EN-US<\/title>\n<meta name=\"description\" content=\"The safety of Brineura\u00ae (cerliponase alfa) was evaluated in 24 patients with CLN2 disease who received at least 1 dose of Brineura in a clinical study with extension of up to 161 weeks.\" \/>\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\/safety\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Safety\" \/>\n<meta property=\"og:description\" content=\"The safety of Brineura\u00ae (cerliponase alfa) was evaluated in 24 patients with CLN2 disease who received at least 1 dose of Brineura in a clinical study with extension of up to 161 weeks.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/hcp.biomarin.com\/en-us\/brineura\/safety\/\" \/>\n<meta property=\"og:site_name\" content=\"BioMarin Brineura HCP EN-US\" \/>\n<meta property=\"article:modified_time\" content=\"2026-03-11T12:51:08+00:00\" \/>\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\/safety\/\",\"url\":\"https:\/\/hcp.biomarin.com\/en-us\/brineura\/safety\/\",\"name\":\"Safety Profile - 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