Brineura – Package Leaflet: InfoCons – Consumer Protection presents, in an accessible format, the official package leaflet of the medicine Brineura, as authorised in the European Union and published in the Union Register of medicinal products.
Before anything else
Read all of this leaflet carefully before you or your child is given this medicine because it contains important information for you.
- Keep this leaflet. You may need to read it again.
- If you have any further questions, ask your doctor.
- If you or your child get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. See section 4.
ℹ️ 1. What Brineura is and what it is used for
Brineura contains the active substance cerliponase alfa, which belongs to a group of medicines known as enzyme replacement therapies. It is used to treat patients wit h neuronal ceroid lipofuscinosis type 2 (CLN2 ) disease, also known as tripeptidyl peptidase-1 (TPP1) deficiency.
People with CLN2 disease do not have any enzyme called TPP1 or they have too little of it and this causes a build -up of substances called lysosomal storage materials. In people with CLN2 disease, these materials build -up in certain parts of the body, mainly the brain.
How Brineura works
This medicine replace s the missing enzyme, TPP1 , which minimises the build -up of the lysosomal storage materials . This medicine works to slow t he progression of the disease.
⚠️ 2. What you need to know before you or your child is given Brineura
You must not receive Brineura
- If you or your child has had life-threatening allergic reactions to cerliponase alfa or any of the other ingredients of this medicine (listed in section 6), and the reactions continue to happen when cerliponse alfa is given again .
- If you or your child has a device implanted to drain ex tra fluid from the brain.
- If you or your child currently has signs of a device infection or problems with the device. Your doctor m ay decide to continue treatment once the device infection or problems are resolved .
Warnings and precautions
Talk to your doctor before you or your child is given Brineura.
- You or your child may get problems with the implanted device used during treatment with Brineura (see section 4 “Possible side effects”) , including infection or a fault in the device.
Signs that you or your child may have an infection include fever, headache, neck stiffness, light sensitivity, nausea, vomiting, and change in mental status . Signs of problems with the device 29 include swelling, redness of the scalp, fluid leaking from device and bulging of the scalp.
Treatment may be interrupted if the device needs to be replaced or until the infection clears.
Within 4 years of use, the access device may need to be replaced and will be determined by your doctor. Talk to your doctor if you have any questions about your device.
- Life-threatening allergic reactions (anaphylactic reactions) are possible with this medicine . Your doctor will monitor you or your child for symptoms of life threatening allergic reactions, such as hives, itching or flushing, swollen lips, tongue, and/or throat, chills, accelerated heart rhythm, shortness of breath, hoarseness, turning blue around finger tips or lips, low muscle tone, fainting , diarrhoea or incontinence . Seek immediate medical care should these symptoms occur.
- Your doctor will check your or your child’s heart rate, blood pressure, respiratory rate, and temperature before, during, an d after treatment. The doctor may decide on a dditional monitoring if it is needed.
- Your doctor will check for abnormal heart electrical activities (ECG) every 6 months. If you or your child has a history of heart problems, your doctor or nurse will monitor your heart activity during each infusion.
- Your doctor may send samples of brain fluid to check for signs of infection.
- This medicine has not been given to patients with advanced disease at the start of treatment or in childre n younger than 1 year of age. Your doctor will discuss whether Brineura treatment is right for you or your child.
Other medicines and Brineura
Tell your doctor if you or your child i s taking, has recently taken, or might take any other medicines.
Pregnancy, breast -feeding and fertility If you are pregnant or breast -feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before treatment with this medicine.
You should not receive this medicine during pregnancy unless clearly necessary. It is not known if this medicine can harm your unborn baby.
You should not receiv e this medicine if you are breast -feeding. It is not known if this medicine passes into human breast milk.
It is not known if this medicine impacts human fertility.
Driving and using machines
It is not known if this medicine will impact the ability to drive or use machines. Please consult your doctor.
Brineura contains sodium and potassium
This medicine contains 17. 4 mg sodium (main component of cooking/table salt) in each vial. This is equivalent to 0.87% of the recommended maximum daily dietary intake of sodium for an adult.
This medicine contains potassium, less than 1 mmol ( 39 mg) per vial, that is to say essentially ‘potassium -free’.
💊 3. How Brineura is given
You or your child will need to have surgery to implant the device for giving this medic ine. The device helps the medicine to reach a specific part of the brain.
This medicine will be given by a doctor with knowledge of giving medicines by intracerebroventricular use (infusion into the fluid of the brain) in a hospital or clinic .
This medicine has not been given to patients younger than 1 year of age or older than 9 years of age (at the start of the clinical trial).
The recommended dose of this medicine is based upon your or your child’s age, and is given once every other week as follows:
- birth to < 6 months: 100 mg
- 6 months to < 1 year: 150 mg
- 1 year to < 2 years: 200 mg (first 4 doses), 300 mg (all other doses)
- ≥ 2 years: 300 mg Your doctor may adjust you or your child’s dose or the amount of time the medicine is given if the infusion is not tolerated, there is an allergic reaction or there is a possible increase of pressure in the brain.
The medicine is slowly pumped through the implanted device. After the medicine has been given, a shorter infusion of a solution is given to flush Brineura out of the infusion equipment so that the full dose reaches the brain . The medicine and solution wil l be given over about 2 to 4 hours and 30 minutes according to your or your child’s dose. Your doctor may lower the dose or the speed of the infusion based on your response during the treatment.
Your doctor may give you or your child medicine s, such as ant ipyretic s to reduce fever or antihistamines to treat allergic reactions before each treatment with this medicine to reduce side effects that can occur during or shortly after treatment .
If you have any further questions on the use of this medicine, ask your doctor.
❗ 4. Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Talk to your doctor or nurse immediately if you experience any of the following:
Very common side effects (may affect more than 1 in 10 people)
- convulsions (seizures)
- reactions during or shortly after being given the medicine, such as hives, itching or flushing, swollen lips, tongue and/or throat, shortness of breath, hoarseness, turning blue around f inger tips or lips , low muscle tone, fai nting or incontinence .
- device-related bacterial infections
Common side effects (may affect up to 1 in 10 people)
- severe allergic reaction (anaphylactic reactions) .
Not known (frequency cannot be estimated from the available data)
- inflammation of the brain (meningitis) due to device -related infection .
This medicine may cause other side effects:
Very common side effects (may affect more than 1 in 10 people)
- fever
- vomiting
- feeling irritable
- headache
- increased or decreased protein in the brain fluid detected by laboratory monitoring
- abnormal results of heart electrical activity (ECG)
- increased cells in the spinal fluid detected by laboratory monitoring
- device does not function correctly due to a blockage detected during preparation for infusion
- leakage of the device 31 – needle issue (infusion needle falls out of implanted device) .
Common side effects (may affect up to 1 in 10 people)
- slower heart beat
- rash
- hives
- device breakage
- device site irritation
- feeling nervous
- disorder of the stomach or intestines .
Not known (frequency cannot be estimated from the available data)
- device is displaced and does not function correctly when preparing for infusion .
Reporting of side effects
If you or your child get s any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. You can also report side effects directly vi a the national reporting system listed in Appendix V . By reporting side effects, you can help provide more inform ation on the safety of this medicine.
🌡️ 5. How to store Brineura
Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date, which is stated on the vials and carton after EXP. The expiry date refers to the last day of that month.
Store upright in a freezer ( -25°C to -15°C). Store in the original package, in order to protect from light.
Transport and distribute frozen ( -85°C to -15°C).
Thawed Brineura and flushing solution should be used immediately. This medicine should only be withdrawn from the unopened vials immediately prior to use. If immediate use is not possible, unopened vials of Brineura or flushing solution should be stored in a refrigerator (2°C – 8°C) and used within 24 hours.
Chemical and physical in -use stability has been demonstrated for up to 12 hours at room temperature (19°C – 25°C ). From a microbiological point of view, open vials or medicinal product held in syringes should be used immediately. If not used immediately, in -use storage times and conditions prior to use are the responsibility of the user.
Your doctor or pharmacist is responsible for storing Brineura. They are also responsible for disposing of any unused Brineura proper ly.
📦 6. Contents of the pack and other information
What Brineura contains
- The active substance is cerliponase alfa. Each vial of Brineura contains 150 mg of cerliponase alfa in 5 ml of solution . Each ml of solution for infusion contains 30 mg of cerliponase alfa.
- The other ingredients of Brineura solution for infusion and the flushing solution are: sodium phosphate dibasic heptahydrate , sodium dihydrogen phosphate monohydrate , sodium chloride, potassium chloride, magnesium chloride hexahydrate, c alcium chloride dihydrate and water for
injections (see section 2 “Brineura contains sodium and potassium ”).
32 What Brineura looks like and contents of the pack Brineura and the flushing solution are solutions for infusion. The Brineura solution for infusion is clear to slightly opalescent, colourless to pale yellow that may occa sionally contain thin translucent fibres or opaque particles. T he flushing solution is clear and colourless .
Pack size: Each pack contains two vials of Brineura solution for infusion and one vial of flushing solution , each containing 5 ml of solution .
Marketing Authorisation Holder and Manufacturer
BioMarin International Limited Shanbally, Ringaskiddy County Cork Ireland
This leaflet was last revised in
This medicine has been authorised under ‘exceptional circumstances’. This means that because of the rarity of this disease it has been impossible to get complete information on this medicine.
The European Medicines Agency will review any new information on this medicine every year and this leaflet will be updated as necessary.
Other sources of information Detailed information on this me dicine is available on the European Medicines Agency web site: http://www.ema.europa.eu . There are also links to other websites about rare diseases and treatments.
📄 Package Leaflet – official source
This article reproduces the information in the official package leaflet, as authorised in the European Union. You can consult the original document here: Product information Brineura (PDF, ec.europa.eu).
You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/17/1192).
⚠️ Important: This article is for information purposes only and does not replace medical advice or the package leaflet inside the medicine box. Read the leaflet carefully before use. For any questions about your treatment, talk to your doctor or pharmacist. If you experience side effects, tell your doctor or pharmacist and report them through your national reporting system.