See below for BioMarin’s United States Terms of Use. For more specific region/country versions, please select from the dropdown menu.

Terms Of Use

Effective Date: October 1, 2018

These Terms of Use (“Terms”) govern your access to and use of the websites and online services that we operate and that link to these Terms, as well as our offline services that reference these Terms (collectively, the “Services”).

Please review these Terms carefully before using the Services because they are a legal contract between you and BioMarin Pharmaceutical Inc. (“BioMarin,” “we,” “us,” or “our”). By using the Services, you agree to be bound by and comply with these Terms. We may change these Terms or modify any features of the Services at any time. The most current version of the Terms can be viewed by clicking on the “Terms of Use” link posted through the Services. You accept the Terms by using the Services, and you accept any changes to the Terms by continuing to use the Services after we post the changes.

Privacy Notice

For more information on how BioMarin processes your personal information, and your rights regarding your personal information, please see our Privacy Notice .

Prohibited Conduct

You may not access or use, or attempt to access or use, the Services to take any action that could harm us or any third party, interfere with the operation of the Services, or in a manner that violates any law(s). For example, and without limitation, you may not:

  • Impersonate any person or entity or falsely state or otherwise misrepresent your affiliation with any person or entity or the origin of any information you provide;
  • Engage in unauthorized spidering, scraping, or harvesting of content or personal information, or use any other unauthorized automated means to compile information;
  • Obtain or attempt to gain unauthorized access to other computer systems, materials, information, or any services available on or through the Services;
  • Use any device, software, or routine to interfere or attempt to interfere with the proper working of the Services or any activity conducted on the Services or attempt to probe, scan, test the vulnerability of, or breach the security of any system or network;
  • Circumvent, reverse engineer, decipher, decompile, disassemble, decrypt, or otherwise alter or interfere with (or attempt, encourage, or support anyone else’s attempt to engage in such activities) any of the software comprising or in any way making up a part of the Services. The use or distribution of tools designed for compromising security (e.g., password guessing programs, cracking tools or network probing tools) is strictly prohibited;
  • Take any action that imposes an unreasonable or disproportionately large load on our network or infrastructure;
  • Upload or otherwise transmit any communication, software, or material that contains a virus or is otherwise harmful to BioMarin’s or its users’ computers or systems;
  • Send or cause to be sent any communication (including e-mail) to other users without their consent (e.g., “mailbombs” or “spamming”);
  • Cause damage, embarrassment, or adverse publicity to BioMarin; or
  • Engage in any other conduct that restricts or inhibits any person from using or enjoying the Services, or that, in our sole judgment, exposes us or any of our users, affiliates, or any other third party to any liability, damages, or detriment of any type.

Violations of system or network security may result in civil or criminal liability. We may investigate and work with law enforcement authorities to prosecute users who violate the Terms. We may suspend or terminate your access to the Services for any or no reason at any time without notice.

Medical Disclaimers

The Services and any information or content provided therein are for informational and educational purposes only and are not a substitute for the professional judgment of a health care professional in diagnosing and treating patients. BioMarin does not give medical advice, nor does it provide medical or diagnostic services. Your reliance upon any content or information provided through the Services is solely at your own risk.

User Content and Communications

You remain fully responsible for the materials that you provide to us, including, without limitation, information, stories, requests, creative works, pictures, photographs, letters, documents, demos, ideas, suggestions, reviews, concepts, methods, systems, designs, plans, techniques, or other materials submitted, posted, uploaded, sent, or otherwise transmitted to us (“User Content”). You agree not to provide User Content that:

  • Infringes on the copyright, trademark, patent, or other intellectual property right of any third party;
  • Is false, misleading, libelous, defamatory, obscene, abusive, hateful, or sexually-explicit;
  • Violates a third party’s right to privacy or publicity;
  • Degrades others on the basis of gender, race, class, ethnicity, national origin, religion, sexual preference, disability, or other classification;
  • Contains epithets or other language or material intended to intimidate or to incite violence;
  • Contains a virus, worm, Trojan Horse, time bomb, or any other harmful program or component;
  • Contains any commercial material or solicits any funds (charitable or commercial), perpetuates chain letters or pyramid schemes, promotes commercial entities, or otherwise engages in commercial activity; or
  • Violates any applicable local, state, national, or international law, or advocates illegal activity.

You may only post User Content that is original and that you have the right to post. By submitting User Content, you grant us a royalty-free, irrevocable, perpetual, non-exclusive, worldwide, fully sublicensable license to publish, reproduce, distribute, display, perform, edit, adapt, modify, translate, create derivative works from, make, sell, export, and otherwise use your User Content (or any portion thereof) in any way that we want and in any form, media, or technology now known or later developed. You hereby waive any moral rights you may have in your User Content. You represent that you have obtained all necessary permissions, including the right to use an individual’s likeness in our advertising and marketing activities, from any individuals identified in or implicated by your submission (including those shown in photographic content), and, in the case of minors, also from their parents or legal guardians, as appropriate.

We are not obligated to publish or use your User Content. BioMarin is not in any manner endorsing the User Content and cannot, and will not, vouch for its reliability. We do not guarantee any confidentiality with respect to any User Content. Please do not send us any confidential or proprietary information or material.

BioMarin is not responsible for any User Content and has no duty to monitor the User Content posted on the Services. You use any information contained in User Content at your own risk.

BioMarin and its designees have the right, in their sole discretion, to monitor, review, edit, remove, delete, disable, refuse, restrict, or terminate access to your User Content or the Services (in whole or in part) at any time, without prior notice and in our sole discretion, for any or no reason. The obligations that you have to us under these Terms shall survive termination of the Services, any use by you of the Services, any User Content on the Services, or these Terms. You will not continue to post any User Content that BioMarin has previously advised you not to post.

Eligibility and Registration

You may be asked to register for certain activities in connection with the Services by creating a user profile. This registration may allow you to participate in interactive features of the Services, such as forums or discussion boards or to enter sweepstakes and contests. When you register, you agree to provide accurate, current, and complete information about yourself as requested or directed on the Services and to promptly update this information to maintain its accuracy. BioMarin has the right to suspend or terminate any account or other registration and to refuse any and all current or future use of the Services if it suspects that such information is inaccurate or incomplete. You are responsible for maintaining the confidentiality of any password and username that you are given or select in connection with the Services, and you are responsible for all activities that occur under your password or account.

Intellectual Property

The Services are protected under the copyright, trademark, and other intellectual property laws of the United States and other countries. All intellectual property rights in the Services are owned by us or our third-party licensors to the full extent permitted under U.S. and international intellectual property laws. Except for content that you have posted on the Services, you may not publish, reproduce, distribute, display, perform, edit, adapt, modify, or otherwise exploit any part of the Services, including the BioMarin name and logo, without our written consent. You may provide a link to the top page of the Services unless and until BioMarin gives notice that you must discontinue linking to the Services.

We respect intellectual property rights. If you believe in good faith that your work has been reproduced or is accessible on the Services in a way that constitutes copyright infringement, please provide our designated agent with the following information in writing:

  • An electronic or physical signature of the person authorized to act on behalf of the owner of the exclusive right that is allegedly infringed;
  • Identification of the copyrighted work or a representative list of the works claimed to have been infringed;
  • Identification of the allegedly infringing material and information reasonably sufficient to permit us to locate the material;
  • Your name, address, telephone number, and email address, so that BioMarin may contact you if necessary;
  • A statement that you have a good faith belief that the disputed use is not authorized by the copyright owner, its agent, or the law; and
  • A statement by you, made under penalty of perjury, that the above information in your notice is accurate and that you are the copyright owner or authorized to act on the copyright owner's behalf.

BioMarin’s designated agent for notice of claims of alleged copyright infringement is:

Eric Davis
Copyright Agent
105 Digital Drive
Novato, CA 94949
Phone: (415) 506-6700
edavis@bmrn.com

If you believe that a user of the Services is a repeat infringer, please contact BioMarin’s designated agent, as described above, and provide enough information for us to verify that the individual is a repeat infringer. Please note that United States law provides significant penalties for falsely submitting a notice of copyright infringement.

Third-Party Content and Links to Third-Party Websites

The Services may contain links to third-party content. We do not control, endorse, sponsor, recommend, or otherwise accept responsibility for such content. Use of any linked third-party content is at the user's own risk.

Disclaimer of Warranties

YOUR USE OF THE SERVICES IS AT YOUR OWN RISK. WE MAKE NO REPRESENTATIONS OR WARRANTIES ABOUT THE OPERATION OF THE SERVCES OR THE INFORMATION, MATERIALS, GOODS, OR SERVICES APPEARING OR OFFERED ON THE SERVICES, ALL OF WHICH ARE PROVIDED "AS IS."

WITHOUT LIMITING THE GENERALITY OF THE FOREGOING, WE DISCLAIM ALL WARRANTIES, EXPRESS OR IMPLIED, INCLUDING, BUT NOT LIMITED TO, ANY (1) WARRANTIES OF MERCHANTABILITY AND FITNESS FOR A PARTICULAR PURPOSE; (2) WARRANTIES AGAINST INFRINGEMENT OF ANY THIRD-PARTY INTELLECTUAL PROPERTY OR PROPRIETARY RIGHTS; (3) WARRANTIES RELATING TO THE TRANSMISSION OR DELIVERY OF THE SERVICES; (4) WARRANTIES RELATING TO THE ACCURACY, RELIABILITY, CORRECTNESS, OR COMPLETENESS OF DATA MADE AVAILABLE ON THE SERVICES OR OTHERWISE BY BIOMARIN; (5) WARRANTIES OTHERWISE RELATING TO PERFORMANCE, NONPERFORMANCE, OR OTHER ACTS OR OMISSIONS BY BIOMARIN OR ANY THIRD PARTY; AND (6) WARRANTY OF TITLE. FURTHER, THERE IS NO WARRANTY THAT THE SERVICES WILL MEET YOUR NEEDS OR REQUIREMENTS OR THE NEEDS OR REQUIREMENTS OF ANY OTHER PERSON.

WE MAKE NO WARRANTIES, EXPRESS OR IMPLIED, (1) THAT THE SERVICES OR ANY EMAIL WE SEND YOU IS FREE OF VIRUSES OR OTHER HARMFUL COMPONENTS THAT MAY INFECT YOUR COMPUTER EQUIPMENT OR OTHER PROPERTY BECAUSE OF YOUR ACCESS TO, USE OF, OR BROWSING ON THE SERVICES OR YOUR DOWNLOADING OF ANY MATERIALS, DATA, TEXT, IMAGES, VIDEO, OR AUDIO FROM THE SERVICES; OR (2) THAT THE SERVICE, WEBSITE CONTENT, FUNCTIONS, OR MATERIALS CONTAINED THEREIN WILL BE TIMELY, SECURE, ACCURATE, COMPLETE, UP-TO-DATE, OR UNINTERRUPTED. BIOMARIN DOES NOT NECESSARILY ENDORSE, SUPPORT, SANCTION, ENCOURAGE, OR AGREE WITH ANY WEBSITE CONTENT OR ANY SUBMITTED MATERIALS, AND WE EXPRESSLY DISCLAIM ANY AND ALL LIABILITY IN CONNECTION WITH USER CONTENT, INCLUDING LINKS TO OR CONTENT CONTAINED ON THIRD-PARTY WEB SITES.

IF APPLICABLE LAW DOES NOT ALLOW THE EXCLUSION OF SOME OR ALL OF THE ABOVE WARRANTIES TO APPLY TO YOU, THE ABOVE EXCLUSIONS WILL APPLY TO YOU TO THE FULLEST EXTENT PERMITTED BY APPLICABLE LAW.

Limitation of Liability

UNDER NO CIRCUMSTANCES, INCLUDING NEGLIGENCE, WILL BIOMARIN, ITS AFFILIATES, OR ANY PARTY INVOLVED IN CREATING, PRODUCING, OR DELIVERING THE SERVICES BE LIABLE FOR DAMAGES OR LOSSES, INCLUDING, BUT NOT LIMITED TO, DIRECT, INCIDENTAL, CONSEQUENTIAL, INDIRECT, SPECIAL, OR PUNITIVE DAMAGES AND LOST PROFITS, ARISING OUT OF YOUR ACCESS, USE, MISUSE, OR INABILITY TO USE THE SERVICES, WEBSITE CONTENT, USER CONTENT, OR ANY LINKED SITES, HOWEVER CAUSED, WHETHER IN CONTRACT, TORT, NEGLIGENCE, STRICT LIABILITY, OR OTHERWISE, EVEN IF BIOMARIN HAS BEEN ADVISED OF THE POSSIBILITY OF SUCH DAMAGES, OR IN CONNECTION WITH ANY FAILURE OF PERFORMANCE, ERROR, OMISSION, INTERRUPTION, DEFECT, DELAY IN OPERATION OR TRANSMISSION, COMPUTER VIRUS, OR LINE OR SYSTEM FAILURE. BIOMARIN WILL NOT BE A PARTY TO, AND WILL HAVE NO RESPONSIBILITY OR LIABILITY FOR, ANY TRANSACTION NEGOTIATED OR ARRANGED BY A USER ARISING IN CONNECTION WITH THE SERVICES. IN THE EVENT THAT YOU HAVE A DISPUTE WITH ANOTHER USER RELATED TO, ARISING FROM, OR IN ANY WAY CONNECTED WITH USE OF THE SERVICES, YOU RELEASE BIOMARIN FROM ANY CLAIMS, DEMANDS, AND DAMAGES OF EVERY KIND AND NATURE ARISING OUT OF OR IN ANY WAY CONNECTED WITH SUCH A DISPUTE. BIOMARIN HAS NO OBLIGATION TO BECOME INVOLVED IN ANY DISPUTE BETWEEN A USER AND ANY OTHER PERSON.

THESE LIMITATIONS APPLY WHETHER THE ALLEGED LIABILITY IS BASED ON CONTRACT, TORT, NEGLIGENCE, STRICT LIABILITY, OR ANY OTHER BASIS, EVEN IF BIOMARIN HAS BEEN ADVISED OF THE POSSIBILITY OF SUCH DAMAGE. BECAUSE SOME JURISDICTIONS DO NOT ALLOW THE EXCLUSION OR LIMITATION OF INCIDENTAL OR CONSEQUENTIAL DAMAGES, BIOMARIN’S LIABILITY IN SUCH JURISDICTIONS SHALL BE LIMITED TO THE EXTENT PERMITTED BY LAW. WITHOUT LIMITING THE GENERALITY OF THE FOREGOING, BIOMARIN DISCLAIMS ALL LIABILITY OF ANY KIND ARISING FROM THE UNAUTHORIZED ACCESS TO OR USE OF YOUR INFORMATION.

IF YOU ARE DISSATISFIED WITH THE SERVICES, YOUR SOLE REMEDY IS TO STOP USING THE SERVICES.

Indemnification

You agree to defend, indemnify, and hold harmless BioMarin and its affiliates, subsidiaries, parent companies, successors, officers, directors, employees, contractors, services providers, and agents from and against any and all demands, claims, damages, liabilities, judgments, losses, costs, expenses, and harms, including, but not limited to, reasonable attorneys’ fees, arising in connection with your use of the Services (including, without limitation, your User Content), online conduct, any violation of these Terms, or dealings or transactions with other persons resulting from use of the Services.

Applicable Law; Jurisdiction

These Terms are governed by, and must be construed in accordance with, the laws of the United States and the State of California, as applicable, without giving effect to their principles of conflicts of law. By using the Services, you waive any claims that may arise under the laws of other countries or territories.

With respect to any and all disputes arising out of or in connection with the Services or these Terms (including, without limitation, the Privacy Notice), BioMarin and you agree to negotiate in good faith and undertake reasonable efforts to cooperate with one another in order to achieve a mutually satisfactory resolution. If you and BioMarin do not resolve any dispute by informal negotiation, any other effort to resolve the dispute will be conducted exclusively by binding arbitration as described in this section. You are giving up the right to litigate (or participate in as a party or class member) all disputes in court before a judge or jury. Instead, all disputes will be resolved before a neutral arbitrator, whose decision will be final except for a limited right of appeal under the Federal Arbitration Act. Any court with jurisdiction over the parties may enforce the arbitrator’s award.

Any proceedings to resolve or litigate any dispute in any forum will be conducted solely on an individual basis. Neither you nor BioMarin will seek to have any dispute heard as a class action or in any other proceeding in which either party acts or proposes to act in a representative capacity. No arbitration or proceeding will be combined with another without the prior written consent of all parties to all affected arbitrations or proceedings.

BioMarin and you agree that all disputes arising under these Terms that cannot be settled through informal negotiation will be settled exclusively through confidential binding arbitration in San Francisco, California, or another forum mutually agreed upon by the parties, pursuant to the Rules of Arbitration (“Rules”) of the International Chamber of Commerce (“ICC”) by a sole arbitrator nominated by agreement of the parties and confirmed in accordance with said Rules. The arbitrator's award shall be binding and may be entered as a judgment in a court of competent jurisdiction. You agree that BioMarin is entitled to obtain preliminary injunctive relief to the extent allowed by law to enforce any of the terms of these Terms pending a final arbitral decision.

To the extent permitted by law, any claim or dispute under this agreement must be filed within one year in an arbitration proceeding. The one-year period begins when the claim or notice of dispute first could be filed. If a claim or dispute isn't filed within one year, it is permanently barred.

If any other provision of this section is found to be illegal or unenforceable, that provision will be severed, with the remainder of this section remaining in full force and effect.

Separate Terms and Conditions

In connection with your use of the Services, you may be asked to consent to policies or terms and conditions in addition to these Terms. Please read these supplemental policies and terms carefully before making any use of such portions of the Services. Any supplemental terms will not vary or replace these Terms regarding any use of the Services, unless otherwise expressly stated.

Access Outside the United States

BioMarin makes no claim that the Services are appropriate for access or use outside the U.S. Your access and use of the Services outside the U.S. are at your own risk, and you are responsible for compliance with the laws of your jurisdiction.

Miscellaneous

These Terms constitute the entire agreement between BioMarin and you, superseding any prior or contemporaneous communications and proposals (whether oral, written or electronic) between you and us. In the event any provision of these Terms is held unenforceable, it will not affect the validity or enforceability of the remaining provisions and will be replaced by an enforceable provision that comes closest to the intention underlying the unenforceable provision. You agree that no joint venture, partnership, employment, or agency relationship exists between you and BioMarin as a result of these Terms or your access to and use of the Services.

Our failure to enforce any provisions of these Terms or respond to a violation by any party does not waive our right to subsequently enforce any terms or conditions of the Terms or respond to any violations. Nothing contained in these Terms is in derogation of our right to comply with governmental, court, and law enforcement requests or requirements relating to your use of the Services or information provided to or gathered by us with respect to such use.

Contact Information

If you believe that someone has violated these Terms, if you have questions regarding these terms, or if you have questions regarding the Services, please notify us by completing the form found here or by telephone at (415) 506-6700.

Indication
Brineura® (cerliponase alfa) injection for intraventricular use 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.
Important Safety Information
Brineura is contraindicated in patients with any sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g. cellulitis or abscess); or suspected or confirmed CNS infection (e.g. cloudy CSF or positive CSF gram stain, or meningitis), any acute intraventricular access device-related complications (e.g., leakage, extravasation of fluid, or device failure), and with ventriculoperitoneal shunts.
Brineura must only be administered via the intraventricular route using aseptic technique to reduce the risk of infection. Prior to each infusion, inspect the scalp for signs of intraventricular access device leakage, failure or potential infection. Brineura is contraindicated if there are acute intraventricular access device-related complications (e.g., leakage, extravasation of fluid, device failure, or bulging of the scalp around or above the intraventricular access device); or sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g. cellulitis or abscess); or suspected or confirmed CNS infection (e.g. cloudy CSF or positive CSF gram stain, or meningitis). Consultation with a neurosurgeon may be needed to confirm the integrity of the device. In case of intraventricular access device complications, discontinue the Brineura infusion and refer to the device manufacturer’s labeling for further instructions. Prior to each infusion of Brineura and when clinically indicated, send cerebrospinal fluid (CSF) samples for testing of cell count and culture.
Material degradation of the intraventricular access device reservoir was reported after approximately 4 years of administration, which may impact the effective and safe use of the device. During benchtop testing such material degradation was recognized after approximately 105 perforations of the intraventricular access device. The intraventricular access device should be replaced prior to 4 years of single-puncture administrations, which equates to approximately 105 administrations of Brineura.
Monitor vital signs before infusion starts, periodically during infusion, and post-infusion in a healthcare setting. Perform electrocardiogram (ECG) monitoring during infusion in patients with a history of bradycardia, conduction disorder, or with structural heart disease. In patients without cardiac abnormalities, regular 12-lead ECG evaluations should be performed every 6 months.
Hypotension was reported in 2 patients during or up to 8 hours after Brineura infusion. Patients did not require alteration in treatment, and reactions resolved spontaneously or after intravenous fluid administration.
One patient experienced hypoxia 8 hours after Brineura infusion, followed by a low mean arterial pressure at 15 hours post infusion. Symptoms resolved after oxygen administration, airway repositioning, and normal saline infusion. One patient reported decreased oxygen saturation, 45 minutes after starting Brineura, with associated low diastolic blood pressures. Hypoxia resolved after oxygen administration. No treatment was administered for the low diastolic blood pressure, which returned to normal while the patient continued to receive Brineura infusion without change to the infusion rate or dose.
Due to the potential for anaphylaxis, appropriate medical support should be readily available when Brineura is administered. If anaphylaxis occurs, immediately discontinue the infusion and initiate appropriate medical treatment. Observe patients closely during and after the infusion.
Hypersensitivity reactions were reported in 11 patients during or within 24 hours after completion of the Brineura infusion. The signs and symptoms observed concomitantly with hypersensitivity reactions include pyrexia, vomiting, pleocytosis, or irritability. Patients were routinely pre-medicated with antihistamines with or without antipyretics or corticosteroids, prior to infusion of Brineura.
The management of hypersensitivity reactions should be based on the severity of the reaction and may include temporarily interrupting the infusion, and/or treatment with antihistamines, antipyretics, and/or corticosteroids. If a severe hypersensitivity reaction occurs, immediately discontinue the infusion and initiate appropriate medical treatment.
Brineura has not been studied in pregnancy or lactation.
Safety and effectiveness in pediatric patients below 3 years of age have not been established.
In clinical trials, the most frequently reported adverse reactions (≥8%) were pyrexia, ECG abnormalities, decreased CSF protein, vomiting, seizures, hypersensitivity, increased CSF protein, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.
Seizures were reported in 12 patients and included atonic, generalized tonic-clonic, focal, and absence. Seizures were managed with standard anticonvulsive therapies and did not result in discontinuation of Brineura treatment.
In clinical studies with Brineura, device-related adverse reactions were reported in 12 patients and included infection, delivery system–related complications, and pleocytosis. Intraventricular access device-related CNS infections were observed in 2 patients; antibiotics were administered, the intraventricular access device was replaced, and treatment continued. Device-related complications did not result in discontinuation of Brineura treatment. Other device-related adverse reactions included 1 patient with leakage of the intraventricular access device and 1 with pleocytosis.
Hematoma adverse reactions were reported in 5 patients and presented as hematoma, post procedural hematoma, traumatic hematoma, and subdural hematoma. Hematomas did not require treatment and did not interfere with Brineura infusion.
Anti-drug antibodies (ADAs) were detected in serum (79%) and CSF (33.3%) in patients treated with Brineura. No association was found between serum or CSF ADA titers and incidence or severity of hypersensitivity.
Intraventricular access device-related infections, including sub-clinical infections and meningitis, have been observed in patients treated with Brineura. The signs and symptoms of infections may not be readily apparent in patients with CLN2 disease. Healthcare providers should be vigilant for the development of signs and symptoms of infection, including meningitis. In clinical studies, antibiotics were administered, the intraventricular access device was replaced, and the patient continued on Brineura treatment.
Inform caregivers of the signs and symptoms of anaphylaxis, hypotension, bradycardia, and device-related complications and meningitis. Instruct them to seek immediate medical care should any of these signs and symptoms occur.
To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088, or go to www.fda.gov/medwatch.
Please see accompanying full Prescribing Information, or visit www.Brineura.com.
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.