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.
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.
| Common Brands | Brineura |
|---|---|
| Drug Class | Enzyme replacement therapy |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
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.
Contact your healthcare provider immediately if you experience any of the following.
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
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.
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.
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.
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.
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.
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.
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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 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 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 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 can help maintain walking or crawling ability for children with this genetic condition.
Yes, the FDA approved Brineura 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 is an example of an orphan drug because it treats a rare medical condition.
Brineura 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 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 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 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 to flush the tubing, needle, and device. This makes sure your child gets the entire dose of Brineura. It also helps prevent any blockages in the device to make sure the line stays open for your child's next infusion.
