VIMIZIM 1 mg /Ml Concentrate For Infusion Solution

Зміст інструкції

1. Vimizim 1 mg/ml concentrate for solution for infusion
   1. What is Vimizim and what it is used for
   2. What you need to know before you use Vimizim
   3. How to use Vimizim
   4. Possible side effects
   5. How to store Vimizim
   6. Content of the pack and other information

Vimizim 1 mg/ml concentrate for solution for infusion

elosulfase alfa
▼ Medicinal product subject to additional monitoring. This will allow the rapid identification of
new safety information. You can contribute by reporting any adverse effects
experienced during the use of this medicine. See the end of section 4 for
information on how to report adverse effects.

Read this leaflet carefully before you use this medicine as it contains important
information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor.
• If you get any side effects, even those not listed in this leaflet, tell your doctor or nurse. See section 4. Contents of this leaflet
• 1. What Vimizim is and what it is used for
• 2. What you need to know before you use Vimizim
• 3. How to use Vimizim
• 4. Possible side effects
• 5. How to store Vimizim
• 6. Contents of the pack and other information

1. What is Vimizim and what it is used for

Vimizim contains an enzyme called elosulfase alfa, which belongs to a group of medicines known
as enzyme replacement therapies. It is used to treat adults and children with
mucopolysaccharidosis Type IVA (MPS IVA, also known as Morquio A syndrome).
People with MPS IVA are completely lacking or have insufficient N-
acetylgalactosamine 6-sulfatase, an enzyme that breaks down specific substances in the body, such as
keratan sulfate, which are found in many tissues of the body, including cartilage and bone. As a
result, these substances are not processed by the body as they should be. They
accumulate in the tissues interfering with their normal function and causing the symptoms of
MPS IVA, such as difficulty walking, breathing problems, short stature and hearing loss.

How Vimizim works
This medicine replaces the natural enzyme N-acetylgalactosamine 6-sulfatase which is missing in
patients with MPS IVA. Treatment has been shown to improve walking ability and reduce
levels of keratan sulfate in the body. This medicine may improve the symptoms of MPS IVA.

2. What you need to know before you use Vimizim

Do not use Vimizim

• if you have had life-threatening allergic reactions to elosulfase alfa or any of the other ingredients of this medicine (listed in section 6)

Warnings and precautions

• If you are treated with Vimizim, you may experience infusion reactions. An infusion reaction is any undesirable effect, including an allergic reaction, that occurs during the infusion or within one day of the infusion (see section 4). If you experience such a reaction, seek immediate medical attention.
• If you have an allergic reaction during the infusion, your doctor may slow down or stop the infusion. Your doctor may also give you additional medicines to control any allergic reactions (e.g. antihistamines and/or corticosteroids).
• If you experience back pain, numbness in your arms or legs, or loss of bladder or bowel control, seek immediate medical attention. These problems may be part of the disease and may be caused by pressure on the spinal cord. Other medicines and VimizimTell your doctor if you are taking, have recently taken or might take any other medicines. Pregnancy, breastfeeding and fertilityYou should not use Vimizim during pregnancy unless it is strictly necessary. It is not known whether Vimizim is excreted in breast milk. Talk to your doctor to assess whether the benefits of taking Vimizim are greater than the possible risk to the newborn during breastfeeding. It is not known whether Vimizim affects human fertility. No effects on fertility have been observed in animals. Driving and operating machineryDizziness has been reported in some patients during Vimizim infusion. Tell your doctor if you have experienced dizziness after the infusion, especially before driving vehicles or using machinery where dizziness can be dangerous. Vimizim contains sodium and sorbitol (E420)This medicine contains 8 mg of sodium (the main component of table salt) per 5 ml vial, equivalent to 0.4% of the maximum daily intake of sodium for an adult. This medicine contains 100 mg of sorbitol per 5 ml vial, equivalent to 40 mg/kg. Sorbitol is a source of fructose. If you (or your child) suffer from hereditary fructose intolerance, a rare genetic disorder, you (or your child) should not take this medicine unless you have first spoken to your doctor. Patients with hereditary fructose intolerance are unable to metabolize fructose, which can cause serious adverse effects. Tell your doctor before taking this medicine if you (or your child) suffer from hereditary fructose intolerance or if your child is no longer able to take sugary foods or drinks because they feel unwell, vomit or have adverse effects such as bloating, stomach cramps or diarrhoea

3. How to use Vimizim

The doctor or nurse will administer Vimizim to you via intravenous infusion.
The medicine must be diluted before administration. The doctor or nurse will
administer medications to you before treatment to reduce allergic reactions and may also
administer others to control fever.

Dosage
The dose received is based on body weight. The recommended dosage regimen for adults and children is
2 mg/kg of body weight administered once a week via an intravenous drip
(intravenous infusion). Each infusion will be administered over approximately 4 hours. Treatment with
Vimizim can be started at a very early age and is intended for long-term use.
If you have any further questions about the use of this medicine, please ask your doctor or nursing staff.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
Side effects have been observed mainly while patients were receiving the medicine
or shortly after (“infusion reactions”). The most serious side effects were severe allergic reactions (observed rarely – may affect up to 1 in 100 people) and mild to moderate vomiting (observed very commonly – may affect more than 1 in 10 people). Symptoms of
an allergic reaction include skin rash, itching or hives (observed commonly –
may affect up to 1 in 10 people). If you experience difficulty swallowing, speaking, severe
difficulty breathing or wheezing, swelling of the face or lips, dizziness or weak pulse; this
may be symptoms of a severe allergic reaction and you should seek immediate medical
attention.Depending on the severity of the side effect, your doctor may slow down or temporarily suspend
the infusion and/or give you other medicines to reduce the effects of severe allergic reactions (e.g. antihistamines and/or corticosteroids) or to reduce fever (antipyretics).
Very common side effects include symptoms of infusion reactions such as headache, nausea,
fever, chills and stomach pain. Other very common adverse reactions were diarrhea, pain in the
mouth and throat, dizziness and difficulty breathing.
Common side effects observed were muscle pain.

Reporting of side effects
If you get any side effects, talk to your doctor or nurse. You can also report side effects directly through
the national reporting system listed in Annex V. By reporting side effects you can help provide more
information on the safety of this medicine.

5. How to store Vimizim

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 pack and vial after
Exp. The expiry date refers to the last day of that month.
Unopened vials:
Store in a refrigerator (2°C–8°C).
Do not freeze.
Store in the original packaging to protect the medicine from light.
Do not use Vimizim if the solution has changed colour or if it contains visible particles.
After dilution:
Use the product immediately once diluted. If not used immediately, the storage times and
conditions during use are the responsibility of the user and must not generally exceed 24 hours between 2°C and 8°C followed by a maximum of 24 hours between 23°C and 27°C
during administration.
Do not dispose of any medicine in waste water or household waste. Ask your pharmacist how to
dispose of medicines you no longer use. This will help protect the environment.

6. Content of the pack and other information

What Vimizim contains

• The active ingredient is elosulfase alfa. Each ml of concentrate contains 1 mg of elosulfase alfa. Each vial of 5 ml contains 5 mg of elosulfase alfa.
• The other ingredients are: sodium acetate trihydrate, sodium dihydrogen phosphate monohydrate, arginine hydrochloride, sorbitol (E420), polysorbate 20 and water for injections (see section 2 in “Vimizim contains sodium and sorbitol (E420)”). Description of the appearance of Vimizim and content of the packVimizim is supplied as a concentrate for infusion solution (sterile concentrate). The concentrate appears from clear to slightly opalescent and from colourless to light yellow and must be free from visible particles. Pack size:1 vial of 5 ml. Marketing authorisation holderBioMarin International Limited Shanbally, Ringaskiddy County Cork, P43 R298 Ireland

Manufacturer
BioMarin International Limited
Shanbally, Ringaskiddy
County Cork, P43 R298
Ireland
More detailed information on this medicinal product is available on the website of the European
Medicines Agency: http://www.ema.europa.eu. Furthermore, links to other websites on rare diseases
and related therapeutic treatments are provided.
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The following information is intended exclusively for healthcare professionals:

Vimizim must not be mixed with other medicinal products in the same infusion, except for

those mentioned previously.
Each vial of Vimizim is for single use only. Vimizim must be diluted with sodium chloride 9 mg/ml (0.9 %) solution for infusion using aseptic technique. The diluted solution of
Vimizim must be administered to patients using an infusion set. An infusion set equipped with a 0.2 µm in-line filter may be used.
Any unused medicinal product and waste materials derived from such medicinal product must be disposed of in accordance with applicable local regulations.

Preparation of the Vimizim infusion (use aseptic technique)

• 1. The number of vials to be diluted must be determined based on the patient's weight and removed from the refrigerator in advance to allow it to reach a temperature between 23°C and 27°C. Do not heat or place the vials in the microwave. The recommended dosage regimen is 2 mg/kg of body weight administered once weekly via an intravenous drip (intravenous infusion). Each infusion will take approximately 4 hours.
• Patient weight (kg) multiplied by 2 (mg/kg) = patient dose (mg)
• Patient dose (mg) divided by 1 (mg/ml Vimizim concentrate) = total number of ml of Vimizim
• Total quantity (ml) of Vimizim divided by 5 ml per vial = total number of vials
• 2. The total number of vials calculated is rounded up to the next whole vial.
• 3. An infusion bag containing sodium chloride 9 mg/ml (0.9 %) solution for infusion, suitable for intravenous administration, is obtained. The total volume of the infusion is determined by the patient's body weight.
• Patients weighing less than 25 kg must receive a total volume of 100 ml.
• Patients weighing 25 kg or more must receive a total volume of 250 ml.
• 4. Before dilution, each vial must be visually inspected for the presence of particulate matter and colour change. The solution should be from clear to slightly opalescent and from colourless to light yellow and must be free from visible particles. Do not shake the vials.
• 5. A volume of sodium chloride 9 mg/ml (0.9 %) solution for infusion equal to the total volume of Vimizim to be added must be withdrawn and discarded from a 100 ml or 250 ml infusion bag.
• 6. The calculated volume of Vimizim is slowly withdrawn from the appropriate number of vials, taking care to avoid excessive shaking.
• 7. The volume of Vimizim must be slowly added to the sodium chloride 9 mg/ml (0.9 %) solution for infusion. When diluted in 100 ml of sodium chloride 9 mg/ml (0.9 %) solution for infusion,the initial rate will be 3 ml/hour. The infusion rate will be increased every 15 minutes as follows: first increase the infusion rate to 6 ml/hour and then increase it progressively in increments of 6 ml/hour every 15 minutes to reach a maximum infusion rate of 36 ml/hour. When diluted in 250 ml of sodium chloride 9 mg/ml (0.9 %) solution for infusion,the initial rate will be 6 ml/hour. The infusion rate will be increased every 15 minutes as follows: first increase the infusion rate to 12 ml/hour and then increase it progressively in increments of 12 ml/hour every 15 minutes to reach a maximum infusion rate of 72 ml/hour.

The infusion rate may be increased as tolerated by patients.

• 8. The diluted solution must be gently mixed before infusion.

• 9. The diluted solution must be visually inspected for particulate matter before use. Do not use if the solution has changed colour or if there is particulate matter in the solution.
• 10. The diluted solution must be used immediately. If not used immediately, the storage times and conditions during use are the responsibility of the user and should generally not exceed 24 hours between 2°C and 8°C followed by a maximum of 24 hours between 23°C and 27°C during administration.