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For assistance, please call 1-866-906-6100

Frequently asked questions

Your Case Manager will guide you every step of the way through your treatment journey, working one-to-one with you to address your unique needs related to treatment. Below are some common questions and answers that might be helpful for you.
BioMarin RareConnections is a program that provides a wide array of support services for BioMarin products to help you understand insurance coverage and gain access to treatment. This may include providing information about financial assistance options and working with your healthcare team to coordinate delivery of BioMarin products for your treatment.
BioMarin RareConnections Case Managers provide families with:
  • Personalized support—individualized, one-to-one assistance to start and continue their BRINEURA® (cerliponase alfa) treatment
  • Financial navigation support—navigating the insurance process and understanding coverage options to gain access to treatment
  • Logistics support—coordinating with a specialty pharmacy for delivery of the medication to the hospital if needed
Learn more about how BioMarin RareConnections can help.
Once you enroll in BioMarin RareConnections, you will be assigned a dedicated experienced Case Manager who understands your unique needs related to access to treatment. Your Case Manager can help you determine your insurance coverage and, if necessary, refer you to financial assistance options for BRINEURA.
To enroll in BioMarin RareConnections and get connected with your Case Manager, start by completing the Patient Authorization Form (PAF).
In order for BioMarin to assist with its medicines and related care, you will need to provide consent to both your healthcare provider and BioMarin:
  • Your healthcare provider needs your written consent to release your protected health information (PHI) to BioMarin
  • BioMarin also needs your written consent to share your information (including genetic information) with other providers to assist you with accessing services that support your treatment
Once you give consent, your healthcare provider will provide BioMarin with the information from your medical records needed to enroll you in BioMarin RareConnections. BioMarin RareConnections will help you work with your insurance provider to get reimbursement for your care and will help you with other case management services.
A patient or an authorized representative for the patient must sign the authorization form.
If you choose not to sign the authorization form, BioMarin will not be able to provide BioMarin RareConnections support in connection with any BioMarin treatment.
BioMarin will use your protected health information (PHI) to enroll you in BioMarin RareConnections, which provides a number of case management services that help support your treatment plan (for example, remind you of appointments or medicine schedules). BioMarin RareConnections will work with you, your healthcare provider, and your health insurance company to help with coverage related to your treatment. If you do not already have insurance coverage, BioMarin RareConnections can help you find insurance options.
The Patient Authorization Form is valid from the date of signature for 10 years, or a shorter time if required by applicable state law, or until you cancel the authorization.
You can cancel the authorization at any time by submitting a written notification to BioMarin RareConnections. Email BioMarin
Connect with a Case Manager by email or call 1-866-906-6100. Once you have been enrolled in BioMarin RareConnections, your dedicated Case Manager will also reach out to you with an introduction and will provide you with their direct contact info. Your Case Manager will be your ongoing partner, providing product support services to help you start and stay on treatment.

Frequently asked questions

Rare diseases can bring uncommon challenges, and everyone’s treatment journey is unique
With BioMarin RareConnections™, you don’t have to face it alone. Your BioMarin team will guide you along your treatment journey, working one-to-one with you to address your unique needs to gain access to treatment. Below are some common questions and answers that might be helpful for you.

About BioMarin RareConnections

BioMarin RareConnections is a program that provides a wide array of support services for BioMarin products to help you understand insurance coverage and gain access to treatment. This may include providing information about financial assistance options and working with your healthcare team to coordinate delivery of BioMarin products for your treatment.

Our dedicated and experienced Case Managers will provide guidance on how you can gain access to BioMarin therapies by:
  • Helping you understand your insurance coverage and financial assistance options
  • Providing you with ongoing product support for BioMarin therapies
  • Coordinating with a specialty pharmacy to deliver your medication at a date and time that is convenient for you
Once you enroll in BioMarin RareConnections, you will be assigned a dedicated experienced Case Manager who understands your unique needs related to treatment. Your Case Manager can help you determine your insurance coverage and, if necessary, identify financial assistance options for your BioMarin treatment.
To enroll in BioMarin RareConnections and get connected with your Case Manager, start by completing the Patient Authorization/Patient Consent Form.
In order for BioMarin to provide product support services, you will need to provide consent to both your healthcare provider and BioMarin:
  • Your healthcare provider needs your written consent to release your protected health information (PHI) to BioMarin
  • BioMarin also needs your written consent to share your information (including genetic testing information) with other providers to assist you with accessing services that support your treatment
Once you give consent, your healthcare provider will provide BioMarin with the information from your medical records needed to enroll you in BioMarin RareConnections. BioMarin RareConnections will help you work with your insurance provider to seek reimbursement for your care and will help you with other case management services.
A patient or an authorized representative for the patient must sign the authorization form.
If you choose not to sign the authorization form, BioMarin will not be able to provide BioMarin RareConnections support in connection with any BioMarin treatment.
BioMarin will use your protected health information (PHI) to enroll you in BioMarin RareConnections, which provides a number of case management services that help support your treatment plan (for example, insurance navigation support). BioMarin RareConnections will work with you, your healthcare provider, and your health insurance company to help with coverage related to your treatment. If you do not already have insurance coverage, BioMarin RareConnections can help you find insurance options.
The Patient Authorization/Patient Consent Form is valid from the date of signature for 10 years, or a shorter time if required by applicable state law, or until you cancel the authorization.
You can cancel the authorization at any time by submitting a written notification to BioMarin RareConnections.

Connect with a Case Manager by email or call 1-866-906-6100. Once you have been enrolled in BioMarin RareConnections, your dedicated Case Manager will also reach out to you with an introduction and will provide you with their direct contact info. Your Case Manager will be an ongoing resource, providing product support services to help you start and continue on treatment.

Indication and Important Safety Information

What is Brineura?

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.

Who should not take Brineura?
  • Patients with active intraventricular access device-related complications (eg, leakage, device failure, or device-related infection, including meningitis)
  • Patients with any sign or symptom of acute or unresolved localized infection around the device insertion site (eg, cellulitis or abscess) or suspected or confirmed central nervous system (CNS) infection (eg, cloudy cerebrospinal fluid [CSF] or positive CSF gram stain, or meningitis)
  • Patients with shunts used to drain extra fluid around the brain

What is the most important information I should know about Brineura?

Administration: 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.

  • Prior to administration, 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

Meningitis and other device-related infections: Intraventricular access device-related infections, including meningitis, were observed with Brineura treatment. Infections required treatment with antibiotics and removal of the access device. 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 child’s doctor should vigilantly be looking for signs and symptoms of infection, including meningitis, during treatment with Brineura
  • Your child’s doctor should inspect the scalp and collect samples of your child’s CSF prior to each infusion of Brineura, to check for infections and that there is no device failure
  • Signs of infection on or around the device insertion site may include redness, tenderness, or discharge

Device-related complications such as device leakage, device failure, extravasation of CSF fluid, or bulging of the scalp around or above the intraventricular access device have occurred. In case of intraventricular access device-related complications, Brineura infusions may be discontinued.

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 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.

Cardiovascular side effects: Low blood pressure and/or slow heart rate may occur during and following the infusion of Brineura. Contact your child’s healthcare provider immediately if these reactions occur. As part of the infusion, the healthcare provider will monitor vital signs (blood pressure, heart rate) before infusion starts, periodically during infusion, and post-infusion, and assess the patient’s status after administration to determine if continued observation may be necessary. Additional monitoring is required for patients with a history of cardiac abnormalities. In patients without cardiac abnormalities, regular 12-lead electrocardiogram (ECG) evaluations should be performed every 6 months.

Hypersensitivity reactions including serious and severe allergic reactions (anaphylaxis) may occur. Symptoms of anaphylaxis may include fever, respiratory distress, rash, vomiting, and irritability, and may occur during treatment or within several hours of Brineura infusion. Seek immediate medical care should signs and symptoms of anaphylaxis occur. Your child may receive medication such as antihistamines before Brineura infusions to reduce the risk of reactions.

If anaphylaxis occurs, you and your child’s healthcare providers should consider the risks and benefits of readministration of Brineura. If the decision is made to readminister Brineura after the occurrence of anaphylaxis, the healthcare providers should ensure appropriately trained personnel and equipment for emergency resuscitation (including epinephrine and other emergency medicines) are readily available during infusion and will start the subsequent infusion at approximately one-half the initial infusion rate at which the anaphylactic reaction occurred.

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, device-related complications, hypersensitivity, collection of blood outside of blood vessels (hematoma), headache, irritability, increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure

The risk information provided here is not comprehensive. Talk to your healthcare provider to learn more or for medical advice about any side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please click here to see full Prescribing Information or visit www.Brineura.com.

Indication and Important Safety Information

What is Brineura?

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.

Who should not take Brineura?
  • Patients with active intraventricular access device-related complications (eg, leakage, device failure, or device-related infection, including meningitis)
  • Patients with any sign or symptom of acute or unresolved localized infection around the device insertion site (eg, cellulitis or abscess) or suspected or confirmed central nervous system (CNS) infection (eg, cloudy cerebrospinal fluid [CSF] or positive CSF gram stain, or meningitis)
  • Patients with shunts used to drain extra fluid around the brain

What is the most important information I should know about Brineura?

Administration: 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.

  • Prior to administration, 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

Meningitis and other device-related infections: Intraventricular access device-related infections, including meningitis, were observed with Brineura treatment. Infections required treatment with antibiotics and removal of the access device. 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 child’s doctor should vigilantly be looking for signs and symptoms of infection, including meningitis, during treatment with Brineura
  • Your child’s doctor should inspect the scalp and collect samples of your child’s CSF prior to each infusion of Brineura, to check for infections and that there is no device failure
  • Signs of infection on or around the device insertion site may include redness, tenderness, or discharge

Device-related complications such as device leakage, device failure, extravasation of CSF fluid, or bulging of the scalp around or above the intraventricular access device have occurred. In case of intraventricular access device-related complications, Brineura infusions may be discontinued.

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 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.

Cardiovascular side effects: Low blood pressure and/or slow heart rate may occur during and following the infusion of Brineura. Contact your child’s healthcare provider immediately if these reactions occur. As part of the infusion, the healthcare provider will monitor vital signs (blood pressure, heart rate) before infusion starts, periodically during infusion, and post-infusion, and assess the patient’s status after administration to determine if continued observation may be necessary. Additional monitoring is required for patients with a history of cardiac abnormalities. In patients without cardiac abnormalities, regular 12-lead electrocardiogram (ECG) evaluations should be performed every 6 months.

Hypersensitivity reactions including serious and severe allergic reactions (anaphylaxis) may occur. Symptoms of anaphylaxis may include fever, respiratory distress, rash, vomiting, and irritability, and may occur during treatment or within several hours of Brineura infusion. Seek immediate medical care should signs and symptoms of anaphylaxis occur. Your child may receive medication such as antihistamines before Brineura infusions to reduce the risk of reactions.

If anaphylaxis occurs, you and your child’s healthcare providers should consider the risks and benefits of readministration of Brineura. If the decision is made to readminister Brineura after the occurrence of anaphylaxis, the healthcare providers should ensure appropriately trained personnel and equipment for emergency resuscitation (including epinephrine and other emergency medicines) are readily available during infusion and will start the subsequent infusion at approximately one-half the initial infusion rate at which the anaphylactic reaction occurred.

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, device-related complications, hypersensitivity, collection of blood outside of blood vessels (hematoma), headache, irritability, increased white blood cell count in the fluid of the brain, device-related infection, slow heart rate, feeling jittery, and low blood pressure

The risk information provided here is not comprehensive. Talk to your healthcare provider to learn more or for medical advice about any side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please click here to see full Prescribing Information or visit www.Brineura.com.