Vimizim – Package Leaflet: InfoCons – Consumer Protection presents, in an accessible format, the official package leaflet of the medicine Vimizim, as authorised in the European Union and published in the Union Register of medicinal products.
Before anything else
ℹ️ 1. What Vimizim is and what it is used for
Vimizim contains an enzyme called elosulfase alfa, which belong s to a group of med icines known as enzyme replacement therapies. It is used to treat adults and ch ildren with mucopolysaccharidosis type IVA (MPS IVA disease, also known as Morquio A Syndrome).
People with MPS IVA disease either lack completely or do not ha ve enough N-acetylgalactosamine-6- sulfatase, an enzyme which breaks down specific substances in t he body such as keratan sulphate, which are found in many tissues of the body, including cartilag e and bone. As a result, these substances do not get broken down and processed by the body as they should. They accumulate in the tissues interfering with their normal function and causing the symptoms of MPS IVA, such as difficulty walking, trouble br eathing, short height, and hearin g loss.
How Vimizim works
This medicine replaces the natural enzyme N-acetylgalactosamine -6-sulfatase which is lacking in MPS IVA patients. Treatment has been shown to improve walking a nd to reduce the levels of keratan sulphate in the body. This medicine may improve the symptoms of MPS IVA.
⚠️ 2. What you need to know before you are given Vimizim
You must not receive Vimizim – if you have experienced life-thre atening allergic reactions to elosulfase alfa or any of the other ingredients of this medicine (listed in section 6).
22 Warnings and precautions
- If you are treated with Vimizim, you may develop infusion react ions. An infusion reaction is any side effect, including an allergic reaction, occurring during t he infusion or within a day following infusion (see section 4). If you experience such a reaction, you should immediately contact your doctor.
- If you have an allergic reaction during your infusion your doct or may slow down, or stop your infusion. Your doctor may also give you additional medicines to manage any allergic reactions (e.g. antihistamines and/or corticosteroids).
- If you experience back pain, numbness in your arms or legs, or lack of control over passing urine or stools , you should immediately contact your doctor . These problems can be a part of the disease and may be caused by pressure on your spinal cord.
Other medicines and Vimizim
Please tell your doctor if you a re taking, have recently taken or might take any other medicines.
Pregnancy, breast-feeding and fertility You should not receive Vimizim during pregnancy unless clearly necessary. It is not known whether Vimizim is excreted in breast mi lk. Discuss with your doctor if the benefits of taking Vimizim are greater than the potential risk to your newborn while breast-fe eding. It is not known if Vimizim impacts human fertility. No eff ect on fertility was observed in animals.
Driving and using machines
Dizziness was reported in some patients during the Vimizim infu sion. Tell your doctor if you feel dizzy after your infusion, especially before driving or using a ny machine where dizziness might be dangerous.
Vimizim contains sodium and sorbitol (E 420) This medicine contains 8 mg sodium (main component of cooking/t able salt) in each 5 ml vial. This is equivalent to 0.4 % of the recommended daily dietary intake of sodium for an adult.
This medicine contains 100 mg sorbitol in each 5 ml vial which is equivalent to 40 mg/kg.
Sorbitol is a source of fructose. If you (or your child) have h ereditary fructose into lerance (HFI), a rare genetic disorder, you (or your child) must not receive this med icine unless discussed with your doctor.
Patients with HFI cannot break down fructose, which may cause s erious side effects.
You must tell your doctor before receiving this medicine if you (or your child) have HFI or if your child can no longer take sweet foods or drinks because they fee l sick, vomit or get unpleasant effects such as bloating, stomach cramps or diarrhoea.
💊 3. How Vimizim is given
Your doctor or nurse will give Vimizim to you by an infusion in to a vein.
The medicine has to be diluted before being given. Your doctor or nurse will give you some medicines before your treatment to reduce allergic reactions and you may also be given medicines to help control any fever.
Dose The dose you receive is based on your body weight. The recomme nded dose regimen for adults and children is 2 mg/kg body weight given once every week through a drip into a vein (intravenous infusion). Each infusion will be given over approximately 4 hou rs. Treatment with Vimizim may be started at as young as an age possible and is intended for long term use.
If you have any further questions on the use of this medicine, ask your doctor or nurse.
❗ 4. Possible side effects
23 Like all medicines, this medicine can cause side effects, altho ugh not everybody gets them.
Side effects were mainly seen wh ile patients were being given t he medicine or shortly after (“infusion reactions”). The most serious side effects were severe allergic reactions (seen uncommonly – may affect up to 1 in 100 people) and mild to moderate vomiting (se en very commonly – may affect more than 1 in 10 people). Symptoms of allergic reaction include ra sh, itching or hives on the skin (seen commonly – may affect up to 1 in 10 people). If you experience any diff iculty swallowing, talking, severe shortness of breath or wheezing, swelling of the face or lips, dizziness or weak pulse; these may represent symptoms of a severe allergic reaction and you should tell your doctor immediately . Based on the severity of the si de effect, your doctor may slo w or temporarily interrupt the infusion and/ or give you additional medicines to reduce th e effects of a severe allergic reaction (e.g. antihistamines and/or cortic osteroids) or to reduce fever (antipyretics).
Very common side effects include symptoms of infusion reactions such as headache, nausea, fever, chills and stomach ache. Other very common adverse reactions w ere diarrhoea, mouth and throat pain, dizziness and trouble breathing.
Common side effects observed was muscle pain.
Reporting of side effects
If you get any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects direct ly via the national reporting system listed in Appendix V . By reporting side effects you can help provide more informati on 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 carton and vial after EXP. The expiry date refers to the last day of that month.
Unopened vials:
Store in a refrigerator (2 C – 8C).
Do not freeze.
Store in the original package in order to protect from light.
Do not use Vimizim if the solution is discoloured or it contain s visible particles.
After dilution:
Once the product has been diluted it should be used immediately . If not used immediately, in-use storage times and conditions are the responsibility of the user and should normally not be longer than 24 hours at 2oC – 8oC followed by up to 24 hours at 23oC – 27oC during administration.
Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will he lp to protect the environment.
📦 6. Contents of the pack and other information
What Vimizim contains
- The active substance is elosulfas e alfa. Each ml of concentrate contains 1 mg elosulfase alfa.
Each vial of 5 ml contains 5 mg elosulfase alfa.
24 – The other ingredients are: sodiu m acetate trihydrate, sodium di hydrogen phosphate monohydrate, arginine hydrochloride, sorbitol, polysorbate 20, and water for in jections (see section 2 under ‘Vimizim contai ns sodium and sorbitol (E420)’).
What Vimizim looks like a nd contents of the pack Vimizim is supplied as a concent rate for solution for infusion (sterile concentrate). The clear to slightly opalescent and colourle ss to pale yellow concentrate m ust be free of visible particles.
Pack sizes: 1 vial of 5 ml Marketing Authorization Holder BioMarin International Limited Shanbally, Ringaskiddy County Cork, P43 R298 Ireland Manufacturer BioMarin International Limited Shanbally, Ringaskiddy County Cork Ireland
This leaflet was last revised in MM/YYYY
Detailed information on this medi cine is available on the Europ ean Medicines Agency web site: http://www.ema.europa.eu . There are also links to other websites about rare diseases and treatments. <————————————————————– ———————————————————-> The following information is int ended for medical or healthcare professionals only:
Vimizim should not be mixed with other medicinal products in the same infusion, except for those mentioned below.
Each vial of Vimizim i s intended for single use only. Vimizim h as to be diluted with sodium chloride 9 mg/ml (0.9 %) solution for infusion using aseptic technique. Diluted Vimizim solution is to be administered to patients using an infusion set. An infusion set equipped with an in-line 0.2 µm filter can be used.
Any unused product or waste material is to be disposed of in ac cordance with local requirements.
Preparation of the Vimizim inf usion (Use aseptic technique) 1. The number of vials to be diluted based on the individual patie nt’s weight must be determined and removed from the refrigerator in advance in order to allow them to reach 23 C – 27C. Do not heat or microwave vials. The recommended dose regimen is 2 mg/kg bo dy weight administered once every week through a drip into a vein (by 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 (m g/ml concentrate of Vimizim) = Total number of ml of Vimizim Total amount (ml) Vimizim divided by 5 ml per vial = Total numb er of vials 2. The calculated total number of vials is rounded up to next whol e vial.
3. An infusion bag containing sodium chloride 9 mg/ml (0.9 %) solu tion for infusion is obtained suitable for intravenous administration. The total volume of th e infusion is determined by the patient’s body weight. Patients weighing less than 25 kg should receive a total volume of 100 ml.
25 Patients weighing 25 kg or more should receive a total volume o f 250 ml.
4. Before dilution, each vial is to be inspected for particulate m atter and discolouration. The clear to slightly opalescent and colourless to pale yellow solution must be free of visible particles. Do not shake vials.
5. A volume of the sodium chloride 9 mg/ml (0.9 %) solution for in fusion is to be withdrawn and discarded from a 100 ml or 250 ml infusion bag equal to the tot al volume of Vimizim to be added.
6. The calculated volume of Vimizim from the appropriate number of vials is slowly withdrawn using caution to avoid excessive agitation.
7. The volume of Vimizim is to be slowly added to the sodium chlor ide 9 mg/ml (0.9 %) solution for infusion.
When diluted with 100 ml sodium chloride 9 mg/ml (0.9 %) soluti on for infusion; the initial rate will be 3 ml/hr. The infusion rate will be increased every 15 minutes as follows: first increase the rate to 6 ml/hr, then increase the rate every 15 minutes by 6 ml/hr increments until a maximum rate of 36 ml/hr is reached.
When diluted with 250 ml sodium chloride 9 mg/ml (0.9 %) soluti on for infusion; the initial rate will be 6 ml/hr. The infusion rate will be increased every 15 minutes as follows: first increase the rate to 12 ml/hr, then increase the rate every 15 minutes b y 12 ml/hr increments until a maximum rate of 72 ml/hr is reached.
Patient weight (kg) Total infusion volume (ml) Step 1 Initial infusion rate 0-15 minutes (ml/hr) Step 2 15-30 minutes (ml/hr) Step 3 30-45 minutes (ml/hr) Step 4 45-60 minutes (ml/hr) Step 5 60-75 minutes (ml/hr) Step 6 75-90 minutes (ml/hr) Step 7 90+ minutes (ml/hr) < 25 1 0 0 3 6 1 21 82 43 0 3 6 ≥ 25 250 6 12 24 36 48 60 72 Infusion rate may be increased as tolerated by patient.
8. The diluted solution is to be mixed gently prior to infusion.
9. The diluted solution is to be vi sually inspected for particulat e matter prior to use. Do not use if the solution is discoloured or if the re is particulate matter in th e solution 10. The diluted solution should be used immediately. If not used im mediately, in-use storage times and conditions are the responsibility of the user and should normal ly not be longer than 24 hours at 2C – 8C followed by up to 24 hours at 23 C – 27C during administration.
📄 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 Vimizim (PDF, ec.europa.eu).
You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/14/914).
⚠️ 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.