Brineura (cerliponase alfa)
An approved treatment for Batten Disease.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Cerliponase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Cerliponase alfa
- Brand name
- Brineura
- Development code
- BMN 190
- Drug class
- Enzyme replacement therapy (intraventricular)
- Manufacturer
- BioMarin Pharmaceutical
- How it's taken
- Infused into the brain's fluid every other week by a doctor experienced in this route.
An enzyme replacement given every other week through a small device placed under the scalp, and the only FDA-approved treatment for CLN2 disease, 1 type of Batten disease. It slows the loss of walking and is approved from birth.
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Where Cerliponase alfa fits
The only FDA-approved treatment for CLN2 disease and for any type of Batten disease. Because it works best before skills are lost, early diagnosis and genetic testing of siblings matter. CLN2 is not part of standard US newborn screening, so families should not assume a baby was tested.
How Cerliponase alfa works
CLN2 disease is caused by changes in the TPP1 gene, so children lack an enzyme that clears waste inside brain cells. The waste builds up, and children lose speech, walking and vision, usually starting between ages 2 and 4. Enzymes given into a vein cannot cross into the brain, so Brineura is infused into the fluid-filled spaces of the brain through a surgically placed reservoir and catheter. It replaces the missing enzyme for CLN2 only and does not treat other types of Batten disease.
Side effects and safety
- Infections. Meningitis and infection of the device have happened. The scalp is checked and a fluid sample is tested before every infusion.
- Device problems. The reservoir and catheter can leak, block or fail, and the reservoir must be replaced before 4 years, about 105 uses.
- Heart effects. Low blood pressure or a slow heart rate can occur. Children with heart problems are monitored during infusions, and others get an ECG every 6 months.
- Infusion reactions. These were more common in children under 3, and the doctor gives an antihistamine and sometimes a fever reducer or steroid beforehand.
- Scalp check and brain fluid test for infection before every infusion
- ECG every 6 months, or during infusions with a history of heart problems
- Device replaced before 4 years
Brineura has a boxed warning for serious allergic reactions, including anaphylaxis, which can happen early or late in treatment. It must not be given with an infection at the device site or in the brain, or with an acute device problem. Children with a shunt that drains brain fluid to the belly cannot receive it. Its warnings also cover meningitis and device infections, device problems, low blood pressure or a slow heart rate, and infusion reactions.
In the first trials, the most common side effects were fever, ECG changes, vomiting, seizures and device problems.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Cerliponase alfa
Infused into the brain's fluid every other week by a doctor experienced in this route. It goes through a reservoir and catheter that a neurosurgeon places at least 5-7 days before the first dose. The full infusion takes about 2-4.5 hours, after a medicine such as an antihistamine. Children 2 and older get 300mg per infusion, and younger children get 100mg under 6 months, 150mg from 6 months to 1 year and 200mg for the first 4 doses between ages 1 and 2, then 300mg.
Availability and cost
Only available as the brand-name product.
Help paying for Brineura
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
A Case Manager helps with insurance and coordinating care, and a Clinical Coordinator supports the family. Enrollment needs a Patient Consent Form and the doctor's enrollment form.
The official page does not say who qualifies. Ask the program. · source - Copay help
Eligible commercially insured patients may pay as little as $0. Not valid with Medicare, Medicaid, VA, DoD, TRICARE or other government programs. Residents of Massachusetts and Rhode Island get help with the drug cost only, not the administration procedure.
For: private insurance · source
Good to know: Call 1-866-906-6100, weekdays 8am-8pm ET. The official page does not describe a free medicine program; ask the Case Manager.
- From a charity · NORD RareCareBatten Disease Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting Applications” - From a charity · NORD RareCareBatten Disease Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year”
Access and eligibility
Approved for children with CLN2 disease, also called TPP1 deficiency, from birth. It is not recommended for babies under 37 weeks (counting pregnancy weeks plus weeks since birth) or under 2.5kg, and it does not treat other forms of Batten disease such as CLN3. Children with a shunt that drains brain fluid to the belly cannot receive it.
Source: BioMarin RareConnections (Brineura support)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
Brineura was compared with children followed in a natural history study, not with placebo, because CLN2 progresses quickly and predictably. In the first trial (NCT01907087), 24 children aged 3-8 were treated. By week 96, 21 of the 22 evaluated had no sustained decline in walking and movement scores. In the natural history group, 21 of 42 did decline. In a later trial (NCT02678689) of 14 children aged 1-6, none had a 2-point decline in motor scores by week 169, against 65% of matched untreated children.
Development history
BioMarin developed cerliponase alfa, and the FDA approved it on April 27, 2017 for children 3 and older with symptoms, the first treatment for any form of Batten disease. On July 24, 2024 the FDA extended it to children from birth, including those without symptoms yet, and added a boxed warning for allergic reactions.
Explore Batten Disease trials
Common questions about Cerliponase alfa
▸What is Cerliponase alfa (Brineura)?
An enzyme replacement given every other week through a small device placed under the scalp, and the only FDA-approved treatment for CLN2 disease, 1 type of Batten disease. It slows the loss of walking and is approved from birth.
▸How does Cerliponase alfa work?
CLN2 disease is caused by changes in the TPP1 gene, so children lack an enzyme that clears waste inside brain cells. The waste builds up, and children lose speech, walking and vision, usually starting between ages 2 and 4. Enzymes given into a vein cannot cross into the brain, so Brineura is infused into the fluid-filled spaces of the brain through a surgically placed reservoir and catheter. It replaces the missing enzyme for CLN2 only and does not treat other types of Batten disease.
▸What are the side effects of Cerliponase alfa?
Brineura has a boxed warning for serious allergic reactions, including anaphylaxis, which can happen early or late in treatment. It must not be given with an infection at the device site or in the brain, or with an acute device problem. Children with a shunt that drains brain fluid to the belly cannot receive it. Its warnings also cover meningitis and device infections, device problems, low blood pressure or a slow heart rate, and infusion reactions. In the first trials, the most common side effects were fever, ECG changes, vomiting, seizures and device problems.
▸How is Cerliponase alfa taken?
Infused into the brain's fluid every other week by a doctor experienced in this route. It goes through a reservoir and catheter that a neurosurgeon places at least 5-7 days before the first dose. The full infusion takes about 2-4.5 hours, after a medicine such as an antihistamine. Children 2 and older get 300mg per infusion, and younger children get 100mg under 6 months, 150mg from 6 months to 1 year and 200mg for the first 4 doses between ages 1 and 2, then 300mg.
▸Is Cerliponase alfa FDA approved?
Yes, Cerliponase alfa (Brineura) is FDA approved (2017) for the treatment of Batten Disease.
▸Does Brineura work for all types of Batten disease?
No, it treats only CLN2 disease, also called TPP1 deficiency. Other types, such as CLN3 juvenile Batten disease, need different treatments, so genetic testing to confirm the type comes first.
▸How is Brineura given?
A neurosurgeon places a small reservoir under the scalp connected to a tube into the brain's fluid. Every other week, the care team infuses Brineura through it over about 2-4.5 hours, after a medicine such as an antihistamine.
▸Can babies without symptoms start Brineura?
Yes, since July 24, 2024 it is approved from birth, including children who have not shown symptoms. In a trial of children aged 1-6, the 7 under age 3 who still walked normally at the start kept their motor scores through week 169. No babies under 1 were in that trial.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2024-08-05. BRINEURA (cerliponase alfa) injection, for intraventricular use: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=485ab3c5-0359-4878-872f-2fe1e7df4047
- U.S. Food and Drug Administration · 2017-04-27. BLA 761052 approval letter. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2017/761052Orig1s000ltr.pdf
- U.S. Food and Drug Administration · 2024-07-24. BLA 761052 supplement S-014 approval letter (from birth). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2024/761052Orig1s014ltr.pdf
- ClinicalTrials.gov. A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease. https://clinicaltrials.gov/study/NCT01907087
- ClinicalTrials.gov. A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease. https://clinicaltrials.gov/study/NCT02678689
- BioMarin. BRINEURA patient support. https://www.biomarin-rareconnections.com/patient/brineura/