Brineura
Brineura (cerliponase alfa) is used for children ages 3 years and older with a specific form of Batten disease (also known as neuronal ceroid lipofuscinosis or NCL). It's the first and only FDA-approved enzyme replacement therapy for this rare genetic condition. The medication is an infusion that's given directly into the fluid around the brain (intraventricular infusion). It's typically given at a facility every other week. Side effects include fever, heart rhythm changes, and vomiting.
What is Brineura (cerliponase alfa)?
What is Brineura (cerliponase alfa) used for?
- Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a type of Batten disease, in children ages 3 years and older
How Brineura (cerliponase alfa) works
People with a specific form of Batten disease called ceroid lipofuscinosis type 2 (CLN2) disease or TTP1 deficiency are missing an enzyme (protein) called TPP1. This enzyme normally breaks down extra or worn-out proteins in the body into smaller pieces to recycle or dispose of them.
When the body is missing TPP1, too much protein builds up in the body, which can lead to symptoms of Batten disease (problems with movement, learning, development, and vision).
Brineura (cerliponase alfa) is an enzyme replacement therapy. It replaces the missing TPP1 in the body.
Drug facts
Side effects of Brineura (cerliponase alfa)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Fever (71%)
- Problems with the electrical activity of the heart (71%)
- Low protein in the fluid of the brain (71%)
- Vomiting (63%)
- Seizures (50%)
- Allergic reaction (46%)
Less Common Side Effects
- Headache
- Irritability
- Feeling jittery
- Bruising
- Slow heart rate
- Low blood pressure
- Device-related infection
Brineura (cerliponase alfa) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious brain infection (meningitis): fever, weakness, chills, trouble breathing, neck pain, neck stiffness, confusion, severe headache
- Serious allergic reaction (anaphylaxis): trouble breathing, tightness in the throat, hives, itching, fast heartbeat, nausea, stomach pain, dizziness, lightheadedness, swelling in the face or tongue
The following Brineura (cerliponase alfa) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Fever, neck pain or stiffness, sensitivity to light, headache, nausea, vomiting, confusion, which may be signs of meningitis
- Low blood pressure—dizziness, feeling faint or lightheaded, blurry vision
- Seizures
- Site infection—skin redness, swelling, warmth, or pain around device site
- Slow heartbeat—dizziness, feeling faint or lightheaded, confusion, trouble breathing, unusual weakness or fatigue
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Headache
- Irritability
- Vomiting
Pros and cons of Brineura (cerliponase alfa)
Pros
- The only enzyme replacement therapy that helps slow the loss of walking or crawling ability in children with CLN2 disease
- Can help maintain children’s ability to walk or crawl over a treatment period of almost 2 years
- Given once every other week
Cons
- Each treatment takes about four and a half hours and must be given by a healthcare provider
- Requires a surgical procedure to insert the access device into the scalp to infuse the medication
- Can raise the risk of infection or heart problems
Pharmacist tips for Brineura (cerliponase alfa)
- Mark your child's appointments on your calendar; appointments should be scheduled for every other week. This helps make sure your child doesn't miss any of their Brineura (cerliponase alfa) infusions.
- The entire Brineura (cerliponase alfa) infusion can take about four and a half hours. Make sure your child gets a good night's sleep, wears comfortable clothes, and has something to do (e.g., a book, tablet, video game) to help pass the time. Also, plan ahead for this time by taking some time off of work or school.
- Your child can potentially have an allergic reaction or infusion-related reaction during or within 24 hours after the Brineura (cerliponase alfa) infusion. This can happen even if they didn't have a reaction to a dose before. Get medical help right away if you notice that your child has any signs or symptoms of an allergic reaction, including trouble breathing or rash.
- The healthcare team might tell you to give your child pretreatment medications before the infusion to prevent or ease any possible allergic reactions from Brineura (cerliponase alfa). These medications can include acetaminophen (Tylenol), diphenhydramine (Benadryl), and a corticosteroid. Pretreatment medications are typically taken 30 minutes to 1 hour before the Brineura (cerliponase alfa) dose.
Common questions about Brineura (cerliponase alfa)
Batten disease is a group of rare genetic problems that happens when there are problems with a specific gene in the body (called a CLN gene). There are 13 different CLN genes, so there are 13 different types of Batten disease. Brineura (cerliponase alfa) is specifically approved for a type of Batten disease called CLN2 disease (also known as TPP1 deficiency). Normally, the CLN2 gene tells the body to make an enzyme called TPP1 to help break down extra or worn-out proteins in the body. But people with CLN2 disease are missing the TPP1 enzyme because of problems with the CLN2 gene. As a result, proteins build up in the body, leading to symptoms such as seizure, vision loss, and movement and speech problems. Brineura (cerliponase alfa) is an enzyme replacement therapy; it replaces missing the TPP1 enzyme in the body so proteins can be properly broken down.
No, there isn't a cure for Batten disease. Brineura (cerliponase alfa) is currently the only available treatment that helps with some symptoms of CLN-2 rather than cure it. More specifically, studies have shown that Brineura (cerliponase alfa) can help maintain walking or crawling ability for children with this genetic condition.
Yes, the FDA approved Brineura (cerliponase alfa) in 2017 for children ages 3 years and older with CLN-2 (a type of Batten disease). It's the first FDA-approved treatment to help slow the loss of walking or crawling ability in children with this rare genetic condition.
Yes, Brineura (cerliponase alfa) is an example of an orphan drug because it treats a rare medical condition.
Brineura (cerliponase alfa) is given as an infusion into the fluid around the brain. This is called an intraventricular infusion. The healthcare team will place a port or access device just below your child's scalp at least 5 to 7 days before the first Brineura (cerliponase alfa) infusion. The device is very small (about the size of a penny), and your child's provider will check to make sure it's not infected or leaking before each infusion.
Brineura (cerliponase alfa) takes four and a half hours to infuse. Giving the infusion faster could be dangerous for your child or could cause a serious allergic reaction. If your child happens to have an allergic reaction with the standard infusion time, the next infusion might need to be given over 9 hours instead. After the Brineura (cerliponase alfa) infusion, your child will get an infusion of electrolytes 's provider will also give them electrolytes to flush the tubing, needle, and device. This adds time to the appointment.
Your child will get an infusion of electrolytes after Brineura (cerliponase alfa) to flush the tubing, needle, and device. This makes sure your child gets the entire dose of Brineura (cerliponase alfa). It also helps prevent any blockages in the device to make sure the line stays open for your child's next infusion.
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Risks and warnings for Brineura (cerliponase alfa)
Brineura (cerliponase alfa) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Device-related complications and infections
Some children had device-related complications while they were treated with Brineura (cerliponase alfa). These complications can include infections like meningitis and port or access device problems. Your child's provider will closely inspect the device before each Brineura (cerliponase alfa) infusion. The healthcare team might also take a sample of the fluid surrounding the brain to test it and make sure there's not an active infection.
To lower the risk of an infection during the infusion, the care team might ask people in the room to wear masks or ask them to step outside the treatment room. Report any signs of an infection or complication to your child's provider right away. These signs might include a high fever; stiff neck; really bad headache with nausea and vomiting; redness, tenderness, or bulging of the scalp; or leaking around the device site.
Heart side effects
- Risk factors: History of slow heart rate or other heart rhythm problem | Structural heart disease
Brineura (cerliponase alfa) can lower blood pressure and heart rate. The care team will check your child's vital signs before, during, and after the Brineura (cerliponase alfa) infusion. The team will also check the electrical activity of the heart through an electrocardiogram (ECG) every 6 months during treatment. The team will monitor your child's heart more closely with ECGs during the infusion if your child has a history of heart problems. Contact your child's provider if your child complains of feeling faint, dizzy, or lightheaded.
Severe allergic reaction
Brineura (cerliponase alfa) can cause serious, sometimes life-threatening allergic reactions. These reactions can happen during or within 24 hours of any infusion, even if your child has safely gotten Brineura (cerliponase alfa) infusions before. Brineura (cerliponase alfa) infusions are given at a facility so that the healthcare team can give your child medical care right away if an allergic reaction happens.
To lower the risk for allergic reactions, the care team might give your child pretreatment medications to take, such as diphenhydramine (Benadryl), acetaminophen (Tylenol), and corticosteroids. If your child has a reaction, the provider might stop or pause the infusion, slow the infusion, or lower the dose of the medication. Get medical help right away if your child has symptoms of a serious allergic reaction, such as trouble breathing, tightness in your throat, hives, itching, and a fast heartbeat.
Brineura (cerliponase alfa) dosage
Typical dosage for Brineura (cerliponase alfa)
The typical dose is 300 mg once every other week, given as an intraventricular infusion (an infusion into the fluid surrounding the brain). The Brineura (cerliponase alfa) infusion is followed by an infusion of electrolytes.
Brineura (cerliponase alfa) contraindications
Contraindications are specific health conditions or situations in which a person should not take a medication due to safety concerns. If you have any of the following conditions or if any of the following apply to you, let your healthcare provider know because Brineura (cerliponase alfa) will not be safe for you to take.
- Active intraventricular access device-related complications (e.g., leaky device)
- Any sign of an infection around the device insertion site or in the brain
- Shunts used to drain extra fluid around the brain
