What is Brineura used to treat?
Brineura ® (cerliponase alfa) 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.
How old do you have to be to take Brineura?
Brineura is approved to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with CLN2 disease. Some words or phrases associated with CLN2 disease may be new to you. See their definitions by clicking on the words in orange, or download the glossary >
How long does Brineura take to work?
It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.4. Brineura treatments will take about 4.5 hours every other week.4 You’ll work with your healthcare team to schedule treatments.
How do you give Brineura?
Brineura is given using an infusion pump. The medicine enters the body through a catheter placed through the skull and into the brain. Brineura is given only by a certain type of healthcare professional. To prevent infection, this medicine is given in a sterile medical setting. This medicine is usually given once every other week.
Does Brineura work?
Brineura in clinical trials Results, published in the New England Journal of Medicine in 2018, showed that Brineura helped children with CLN2 disease maintain their ability to walk or crawl over two years.
What does Brineura do?
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.
How much does Brineura cost?
Most patients are on federal healthcare programs, the company says. Brineura will carry a list price of $27,000 per carton, with patients requiring one carton every other week, according to BioMarin execs. That's a total of $702,000 per year before any discounts.
What are treatments for Batten disease?
The only treatment approved by the U.S. Food and Drug Administration to treat Batten disease is Brineura (cerliponase alfa), an enzyme replacement therapy designed to slow the loss of walking ability in children with a type of Batten disease called CLN2.
Who makes Brineura?
San Rafael, Calif. -based BioMarin Pharmaceutical announced that the U.S. Food and Drug Administration (FDA) approved Brineura (cerliponase alfa) for rare disease late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), which is also called tripeptidyl peptidase 1 (TPP1) deficiency.
What is CLN2 Batten disease?
CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), which may also be collectively referred to as Batten disease. All these disorders affect the nervous system and typically cause worsening problems with vision, movement, and thinking ability.
What is the life expectancy of Batten disease?
Children with Batten disease have a greatly shortened life expectancy. Children with infantile Batten disease often die in early childhood. Children with later onset forms of the disease may live into their teens to thirties, while those who develop the disease in adulthood may have a normal life expectancy.
Is Batten disease curable?
There is no cure for these disorders but a treatment for one of the forms (CLN2 disease) has been approved by the U.S. Food and Drug Administration (see Treatment section). Children with all forms of Batten disease have a greatly shortened life expectancy.
Is Batten disease always fatal?
Over time, affected children suffer mental impairment, worsening seizures, and progressive loss of sight and motor skills. Eventually, children with Juvenile Batten Disease become blind, bedridden, and unable to communicate. Juvenile Batten Disease is always fatal by the late teens or twenties.
How often is brineura given?
Brineura is administered every other week through intraventricular infusion. 1 This method allows Brineura to be delivered directly to a ventricle in the brain, and then into the fluid surrounding the brain, known as the cerebrospinal fluid (CSF). Brineura is delivered into the CSF to help reach cells that are affected by CLN2 disease. Knowledgeable members of your healthcare team will give your child’s Brineura infusions1 .
What is a brineura treatment plan?
HOSPITAL TREATMENT PLAN. Brineura is a unique therapy , so creating a treatment plan specific to your child’s needs may take some time. Brineura requires a multidisciplinary team—that means many people from different departments in the hospital will be involved.
How long does it take to get a Brineura implant?
BRINEURA INFUSIONS CAN BEGIN. It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted. 1 Work with your healthcare team to schedule treatments. Brineura treatment will take about 4.5 hours every other week 1. MRI, magnetic resonance imaging.
Does the hospital reimburse for Brineura?
The hospital will work with your insurance provider to establish reimbursement for Brineura therapy. As a caregiver, you’re the most important part of your child’s team. You are your child's advocate and a key source of information, whether it’s medical records or insights into your child's well-being.
Is brineura right for kids?
Your doctor will determine if Brineura is right for your child. DOCTOR’S APPOINTMENT. You’ll consult with your child’s doctor and, together, determine whether Brineura is right for your child. Your child’s doctor may consult with other experts more familiar with treating CLN2 disease.
What is brineura treatment?
About Brineura - Brineura. Brineura (cerliponase alfa) is the only treatment that directly addresses the cause of CLN2 disease by helping to replace the TPP1 enzyme. What is Brineura?
What is the only treatment for CLN2?
Brineura® (cerliponase alfa) is the only treatment that directly addresses the cause of CLN2 disease by helping to replace the TPP1 enzyme.
How is brineura administered?
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.
What is Brineura approved for?
Brineura is approved to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with CLN2 disease. Some words or phrases associated with CLN2 disease may be new to you. See their definitions by clicking on the words in orange, or download the glossary >. home-pic01.
What is brineura therapy?
Brineura® (cerliponase alfa) is the only enzyme replacement therapy that helps treat CLN2 disease, a common form of Batten disease. Brineura is approved to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age ...
How long did Brineura help children walk?
Brineura helped maintain children’s ability to walk, with or without assistance, over approximately 2 years of treatment. Learn more about Brineura study results >. Discover the path toward treatment with Brineura. Every child’s journey with Brineura will be different. Learn more about how to get started >.
What are the symptoms of Brineura?
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.
What are the signs of infection on a brineura?
Signs of infection on or around the device insertion site may include redness, tenderness, or discharge.
What is the only enzyme replacement therapy that helps treat CLN2 disease?
Helping make more steps possible by slowing loss of walking ability. Brineura® (cerliponase alfa) is the only enzyme replacement therapy that helps treat CLN2 disease, a common form of Batten disease.
How to use brineura?
Use aseptic technique when preparing the Brineura syringe for infusion. Label one sterile syringe “Brineura” and attach the syringe needle. Remove the green flip-off caps from the two Brineura vials. Use the “Brineura” labeled syringe to withdraw a total of 10 mL from the Brineura vials. Do not dilute Brineura.
What is Brineura used for?
Brineura 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.
How long after Brineura infusion does hypotension occur?
In the clinical studies, hypotension was reported in 2 (8%) patients, which occurred during or up to eight hours after Brineura infusion. Patients did not require alteration in treatment, and reactions resolved spontaneously or after intravenous fluid administration [see Adverse Reactions ( 6.1 )].
What is the difference between brineura and intraventricular electrolytes?
Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Brineura is a clear to slightly opalescent and colorless to pale yellow solution. Intraventricular Electrolytes is a clear to colorless solution.
How long after implantation of Brineura?
The intraventricular access device must be implanted prior to the first infusion. It is recommended that the first dose be administered at least 5 to 7 days after device implantation. Brineura is intended to be administered with the B Braun Perfusor® Space Infusion Pump System (Product Code: 8713030U).
How many patients have hypersensitivity reactions to Brineura?
In clinical trials, a total of 11 (46%) patients experienced hypersensitivity reactions during the infusion or within 24 hours of completion of the infusion. Patients in clinical trials were routinely pre-medicated with antihistamines with or without antipyretics or corticosteroids, prior to infusion of Brineura.
Can you administer brineura vials?
Brineura and the Intraventricular Electrolytes must only be administered by the intraventricular route, using the provided Administration Kit for use with Brineura. Each vial of Brineura and Intraventricular Electrolytes is intended for a single dose only.
How is brineura given?
Brineura is given using an infusion pump. The medicine enters the body through a catheter placed through the skull and into the brain. Brineura is given only by a certain type of healthcare professional. To prevent infection, this medicine is given in a sterile medical setting.
What is brineura used for?
What is Brineura? Brineura is used to slow the loss of ability to crawl or walk in children with symptoms of a rare genetic condition called ceroid lipofuscinosis type 2 disease (CLN2). Brineura is for use in children who are at least 3 years old.
How long does it take for a child to get electrolytes after cerliponase alf
Your child will also receive an infusion of electrolytes after each infusion of cerliponase alfa. The entire procedure will take about 4.5 hours to complete. About 30 to 60 minutes before each Brineura infusion, your child will be given medication to prevent certain side effects of this medicine.
What are the complications of a Brineura catheter?
complications with the surgically implanted catheter used to deliver Brineura; any sign of infection on the child's scalp in the area around the implanted catheter; or. a shunt in the brain (to help drain fluid build-up around the brain). heart rhythm problems.
How long does it take for a baby to get a reaction to brineura?
Brineura side effects. Get emergency medical help if your child has signs of an allergic reaction within 24 hours after each infusion. Symptoms may include fever, vomiting, fussiness, hives, difficult breathing, and swelling in the face or throat.
How often should a child be monitored for oxygen?
In some cases, the child's heart function should be checked every 6 months.
Is brineura good for ClN2?
Brineura may help slow the loss of certain physical abilities in children with ceroid lipofuscinosis type 2 disease (CLN2). However, this medicine is not a cure for this condition. Brineura may also be used for purposes not listed in this medication guide.
How often should I take brineura?
The recommended dosage of Brineura in pediatric patients 3 years of age and older is 300 mg administered once every other week by intraventricular infusion. Administer Brineura first followed by infusion of the Intraventricular Electrolytes each at an infusion rate of 2.5 mL/hr. The complete Brineura infusion, including the required infusion of Intraventricular Electrolytes, is approximately 4.5 hours.
Can you administer brineura vials?
Brineura and the Intraventricular Electrolytes must only be administered by the intraventricular route, using the provided Administration Kit for use with Brineura. Each vial of Brineura and Intraventricular Electrolytes is intended for a single dose only.
How is brineura given?
Brineura is only given by infusion into the fluid of the brain (known as an intra ventricular 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.
What should a doctor do before a Brineura infusion?
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.
What is Brineura used for?
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.
What is the purpose of brineura?
Brineura is the first enzyme replacement therapy to be directly administered into the fluid of the brain, treating the underlying cause of CLN2 disease by helping to replace the deficient TPP1 enzyme missing in affected children.
How long after Brineura infusion can you have fever?
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.
What happens if you give your child an infusion?
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.
How to report side effects of BioMarin?
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.
Indications and Usage
Dosage and Administration
- Important Preparation and Administration Information
1. Aseptic technique must be strictly observed during preparation and administration. 2. Brineura should be administered by, or under the direction of, a physician experienced in intraventricular administration. 3. Prior to each infusion of Brineura, inspect the scalp for signs of intraventricula… - Dosage
The recommended dosage of Brineura in pediatric patients 3 years of age and older is 300 mg administered once every other week by intraventricular infusion. Administer Brineura first followed by infusion of the Intraventricular Electrolytes each at an infusion rate of 2.5 mL/hr. The complet…
Dosage Forms and Strengths
- Injection: Brineura 150 mg/5 mL (30 mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5 mL in a single-dose vial. Brineura is a clear to slightly opalescent and colorless to pale yellow solution. Intraventricular Electrolytes is a clear to colorless solution [see How Supplied/Storage and Handling (16)].
Contraindications
- Brineura is contraindicated in patients with: 1. 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) [see Warnings and Precautions (5.1)]. 2. any acute intraventricular access device-related complication (e.g., lea…
Warnings and Precautions
- Meningitis and Other Intraventricular Access Device-Related Infections
Bacterial meningitis requiring antibiotic treatment and removal of the device was reported during postmarketing use of Brineura. Additionally, in clinical trials and during postmarketing use there were reports of other device-related clinical infections which were confirmed by positive CSF cul… - Intraventricular Access Device-Related Complications
During the clinical trial and in postmarketing reports, intraventricular access device-related complications were reported (e.g., device leakage, device failure extravasation of CSF fluid, or bulging of the scalp around or above the intraventricular access device) [see Adverse Reactions …
Adverse Reactions
- The following adverse reactions are described below and elsewhere in the labeling: 1. Meningitis and Other Intraventricular Access Device-Related Infections [see Warnings and Precautions (5.1)] 2. Intraventricular Access Device-Related Complications [see Warnings and Precautions (5.2)] 3. Cardiovascular Adverse Reactions [see Warnings and Precautions (5.3)] 4. Hypersensitivity Reac…
Use in Specific Populations
- Pregnancy
Risk Summary There are no available data on Brineura use in pregnant women to inform a drug-associated risk of pregnancy-related outcomes. Animal reproduction studies have not been conducted using cerliponase alfa. The estimated background risk of major birth defects and mis… - Lactation
Risk Summary There are no data on the presence of cerliponase alfa in human milk, the effects on the breastfed child, or the effects on milk production. The lack of clinical data during lactation precludes a clear determination of the risk of Brineura to an infant during lactation; therefore, th…
Description
- Cerliponase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. The active substance is a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase. The primary activity of the mature enzyme is the cleavage of N-terminal tripeptides from a broad range of protein substrates. Cerliponase alfa co…
Clinical Pharmacology
- Mechanism of Action
CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. TPP1 has no known substrate specificity. Deficiency in TPP1 activity results in the accumulation of lysosomal storag… - Pharmacokinetics
The pharmacokinetics of cerliponase alfa were evaluated in patients with CLN2 disease who received intraventricular infusions of 30 mg (0.1 times the approved recommended dosage), 100 mg (approximately 0.3 times the approved recommended dosage), and 300 mg over approximat…
Nonclinical Toxicology
- Carcinogenesis, Mutagenesis, Impairment of Fertility
Carcinogenicity, genotoxicity, and fertility studies have not been performed with cerliponase alfa. Based on the mechanism of action, cerliponase alfa is not expected to be tumorigenic.