150 mg infusion solution 
cerliponas alpha

1. What Brineura is and what it is used for

Brineura contains the active substance cerliponas alfa, which belongs to a group of medicines called enzyme replacement therapies. It is used to treat patients with the disease neuronal ceroid lipofuscinoses type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.

People with CLN2 disease lack, or have too little of, an enzyme called TPP1. This causes an accumulation of substances called lysosomal storage materials. In people with CLN2 disease, these materials accumulate in certain parts of the body, mainly in the brain.

How Brineura works

This drug replaces the missing enzyme TPP1, which minimizes the accumulation of lysosomal storage materials. This medicine works to slow down the course of the disease.

2. What you need to know before you or your child get Brineura

You must not use Brineura:

  • if you or your child have had life-threatening allergic reactions to cerliponas alfa or any of the other ingredients of this medicine (listed in section 6) and the reaction continues to occur when cerliponas alfa is given again.
  • if you or your child has a tube implanted to drain excess fluid from the brain.
  • if you or your child currently have signs of an infection that may be due to the implanted device being used during treatment, or if there is a problem with the device. The doctor may decide to continue treatment once the device-related infection or problems have been resolved.

Warnings and cautions

Talk to a doctor before you or your child get Brineura.

  • You or your child may experience problems with the implanted device used during treatment with Brineura (see section 4 “Possible side effects”), including infection or a defect in the device. Signs that you or your child may have an infection include fever, headache, stiff neck, photosensitivity, nausea, vomiting, and altered state of mind. Signs of problems with the device include swelling, redness of the scalp, fluid leakage from the device, and bulging of the scalp. Treatment may be interrupted if the device needs to be replaced or until the infection is was gone. After prolonged use, the access unit may need to be replaced, as determined by your doctor. Talk to your doctor if you have any questions regarding your device.
  • Life-threatening allergic reactions are possible with Brineura. Your doctor will monitor you or your child for symptoms of life-threatening allergic reactions: hives, itching or redness, swollen lips, tongue and/or throat, chills, increased heart rate, shortness of breath, hoarseness, turning blue around fingertips or lips, low muscle tone, fainting, diarrhea or incontinence. Seek medical attention immediately if these symptoms occur.
  • Your doctor will check your or your child’s heart rate, blood pressure, respiratory rate, and temperature before, during, and after treatment. The doctor may decide to perform additional checks if necessary.
  • The doctor will check every 6 months for abnormal electrical activity in the heart ( ECG ). If you or your child have had heart problems in the past, your doctor or nurse will monitor your heart activity during each infusion.
  • The doctor should send samples of the cerebrospinal fluid for checking for signs of infection.
  • Brineura has not been given to patients with signs of advanced disease at the start of treatment or to children under 2 years 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 are taking, have recently taken, or might take any other medicines.

Pregnancy, breastfeeding, 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 taking this medicine.

You should not be treated with Brineura during pregnancy unless clearly necessary. It is unknown whether Brineura is excreted in human milk. Breast-feeding should be discontinued during treatment with Brineura. It is unknown whether Brimeura affects the ability to have children.

Driving and using machines

It is not known whether Brineura affects the ability to drive and use machines. Consult your doctor.

Brineura contains sodium:

Each vial contains 44 mg of sodium. This should be considered by patients on a controlled sodium diet.

3. How to get Brineura

You or your child will need surgery to implant the device to get Brineura. The device helps the drug reach a specific part of the brain.

Brineura is given by a doctor who is knowledgeable in administering drugs intracerebroventricularly ( infusion into the fluid-filled cavities of the brain) in a hospital or clinic.

Brineura has not been given to children younger than 2 years or to children older than 8 years (at the start of the clinical trial). There is limited experience in some patients who are 2 years old.

The recommended dose of Brineura is based on your or your child’s age and is given once every two weeks as follows:

  • 0 to less than 6 months: 100 mg
  • 6 months to less than 1 year: 150 mg
  • 1 year to younger than 2 years: 200 mg (first 4 doses), 300 mg (all other doses )
  • 2 years and older: 300 mg

Your doctor may adjust your or your child’s dosage or infusion time for the medicine if the body does not tolerate an infusion, if an allergic reaction occurs or if there are signs of a possible increase in pressure in the brain.

The drug is slowly pumped through the implanted device. Once the drug has been given, a shorter infusion is given with a solution to flush Brineura out of the infusion kit so that the full dose reaches the brain. The medicine and the solution are given for about 2 to 4 hours, depending on the dose to be given. Your doctor may reduce your dose or infusion rate based on how the treatment works.

Your doctor may give you or your child antipyretics, or antihistamines to treat allergic reactions, before each Brineura treatment to reduce the side effects that occur during or shortly after treatment.

If you have any further questions on the use of this product, ask your doctor.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Talk to a doctor or nurse immediately if you experience any of the following:

Very common side effects (may affect more than 1 in 10 people):

  • fever
  • vomiting
  • irritability
  • seizures (seizures)
  • reactions during or shortly after the medicine have been given, such as hives, itching or redness, swollen lips, tongue and/or throat, shortness of breath, hoarseness, turning blue around fingertips or lips, low muscle tone, fainting or incontinence

Common side effects (may affect up to 1 in 10 people):

  • the bacterial infection you associated with the implanted device
  • slower pulse
  • the device does not work properly due to a blockage discovered during the preparation of the infusion, a
  • severe allergic reaction

No known frequency (can not be calculated based on available data)

  • unit is moved and does not work properly in the preparation of the infusion, a
  • inflammation of the brain due to device-related infection

This medicine may cause other side effects:

Very common side effects:

  • headache
  • increased or decreased protein content in the cerebrospinal fluid
  • abnormal ECG
  • increased number of cells in the spinal fluid which is detected by laboratory tests
  • infection of the nose or throat (cold)
  • needle problems (the infusion needle detaches from the implanted device)

Common side effects:

  • pain
  • rash
  • hives
  • head lowering (so that the chin falls down towards the chest)
  • magont
  • leakage of the device
  • blisters in the mouth or on the tongue
  • swelling or redness of the eyelids and the white part of the eye
  • nervousness
  • disorders of the stomach and intestines

Reporting of side effects

If you get any side effects, talk to your doctor, pharmacist, or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly to the Medical Products Agency, www.lakemedelsverket.se. By reporting side effects, you can help increase drug safety information.

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 pack after EXP. The expiration date is the last day of the specified month.

Store upright in a frozen state (-25 ° C to -15 ° C). Store and transport froze (-85 ° C to -15 ° C). Store in the original package. Sensitive to light.

Thawed Brineura and rinse solution should be used immediately. The product should only be withdrawn from unopened vials immediately before use. If immediate use is not possible, keep unopened vials of Brineura and rinsing solution stored at 2-8 ° C and use within 24 hours.

Chemical and physical in-use stability has been demonstrated for up to 12 hours at room temperature (19-25 ° C). From a microbiological point of view, opened vials or medicines contained 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.

Doctors or pharmacists are responsible for the storage of Brineura. They are also responsible for the correct disposal of any unused Brineura.

6. Contents of the packaging and other information

Content declaration

  • The active substance is cerliponas alfa. 1 vial of Brineura contains 150 mg of cerliponase alfa in 5 ml solution. 1 ml solution for infusion contains 30 mg cerliponase alfa.
  • The other ingredients in Brineura and the flushing solution are: sodium phosphate dibasic heptahydrate, sodium dihydrogen phosphate monohydrate, sodium chloride, potassium chloride, magnesium chloride hexahydrate, calcium chloride dihydrate and water for injections (see section 2 “Brineura”).

What the medicine looks like and the contents of the pack

Brineura and the rinsing solution are solutions for infusion. The solutions are clear to slightly opalescent, colorless to pale yellow. The brineura solution may sometimes contain thin transparent fibers or opaque particles.

Pack size: 3 vials (two vials of Brineura and one vial of rinse solution), each vial contains 5 ml of solution.

Marketing Authorization Holder and Manufacturer

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork

Ireland

Muhammad Nadeem

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