Patient leaflet - Naglazyme 1 mg/ml concentrate for solution for infusion
1. What this medicine is and what it is used for
Naglazyme is used to treat patients with MPS VI disease (Mucopolysaccharidosis VI).
People with MPS VI disease have either a low level, or no level, of an enzyme called N-acetylgalactosamine 4-sulfatase, which breaks down specific substances (glycosaminoglycans) in the body. As a result, these substances do not get broken down and processed by the body as they should. They accumulate in many tissues in the body, which causes the symptoms of MPS VI.
How this medicine works
This medicine contains a recombinant enzyme called galsulfase. This can replace the natural enzyme which is lacking in MPS VI patients. Treatment has been shown to improve walking and stair-climbing ability, and to reduce the levels of glycosaminoglycans in the body. This medicine may improve the symptoms of MPS VI.
2. What you need to know before you are given this medicine
You must not receive this medicine
If you have experienced severe or life-threatening allergic (hypersensitive) reactions to
galsulfase or any of the other ingredients of Naglazyme and re-administration of the medicine was not successful.
Warnings and precautions
If you are treated with Naglazyme, you may develop infusion-associated reactions. An
infusion associated reaction is any side effect occurring during the infusion or until the end of the infusion day (see section 4 “Possible Side Effects”). When you experience such a reaction, you should immediately contact your doctor.
If you have an allergic reaction your doctor may slow down, or stop, your infusion. Your
doctor may also give you additional medicines to manage any allergic reactions. - If you have a fever, or if you are having difficulty breathing before this medicine is given, talk
with your doctor about delaying your Naglazyme infusion.
If you have an underlying heart condition, please inform your doctor at any point while
being treated with Naglazyme. They may adjust your infusion based on this information. - This medicine has not been tested in patients with kidney or liver problems. Talk to your
doctor if you have kidney or liver insufficiency.
Please talk to your doctor if you experience muscle pain, numbness in your arms or legs, or
any bowel or bladder problems as these may be caused by pressure on your spinal cord.
Other medicines and Naglazyme
Please tell your doctor if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.
Pregnancy and breast-feeding
Naglazyme should not be given during pregnancy unless clearly necessary. Ask your doctor or pharmacist for advice before taking any medicine. It is not known whether galsulfase is excreted in milk, therefore breast-feeding should be stopped during Naglazyme treatment. Ask your doctor or pharmacist for advice before taking any medicine.
Driving and using machines
No studies on the effects on the ability to drive and use machines have been performed.
This medicine contains Sodium
Each 5 ml vial contains 0.8 mmol (18.4 mg) of sodium and is administered in sodium chloride 9 mg/ml solution for injection. To be taken into consideration by patients on a controlled sodium diet.
3. How this medicine is given
Your doctor or nurse will administer Naglazyme to you.
The dose you receive is based on your body weight. The recommended dose is 1 mg/kg body weight administered once every week through a drip into a vein (by intravenous infusion). Each infusion will take approximately 4 hours. For the first hour the infusion rate will be slow (approximately 2.5% of the total solution), with the remaining volume (approximately 97.5%) being taken over the next 3 hours.
If you are given more Naglazyme than you should
Naglazyme is administered under the supervision of a nurse or doctor, he or she will check that the correct dose has been given and act accordingly if necessary.
If you forget to take this medicine
If you have missed a Naglazyme infusion, please contact your doctor. 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.
Side effects were mainly seen while patients were being given the medicine or shortly after (“infusion associated reactions”). The most serious side effects were swollen face and fever (very common); longer than normal gaps between breaths, difficulty breathing, asthma and hives (common); and swelling of the tongue and throat, and serious allergic reaction to this medicine (unknown frequency).
If you experience any reaction like this, please tell your doctor immediately. You may need to be given additional medicines to prevent an allergic reaction (e.g. antihistamines and/or corticosteroids) or to reduce fever (antipyretics).
The most common symptoms of infusion associated reactions include fever, chills, rash, hives and shortness of breath.
Very common side effects (these may affect more than 1 in 10 people):
- Sore throat
- Gastroenteritis
- Poor reflexes
- Headache
- Inflammation of the eye
- Cloudy eyes
- Poor hearing
High blood pressure
- Nasal congestion
- Bulging belly button
- Vomiting
- Nausea
- Itching
- Pain (including ear, abdominal, joint, chest pain)
- Malaise
Common side effects (these may affect up to 1 in 10 people):
- Tremor
- Low blood pressure
Cough
- Wheezing
- Skin redness
Other side effects with an unknown frequency:
- Shock
- Tingling
- Decreased heart rate
Increased heart rate
- Bluish skin
- Skin paleness
- Low blood-oxygen
- Rapid breathing
Reporting of side effects
If you get 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 (see details below). By reporting side effects you can help provide more information on the safety of this medicine.
United Kingdom
Yellow Card Scheme
Website: www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store
5. How to store this medicine
Keep out of the sight and reach of children.
Do not take use this medicine after the expiry date which is stated on the 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.
Diluted solutions:
Chemical and physical in-use stability has been demonstrated for up to 4 days at room temperature (23°C - 27°C).
From a microbiological safety point of view, the product is to be used immediately. If not used immediately, in-use storage times and conditions are the responsibility of the user and must normally not be longer than 24 hours at 2°C - 8°C followed by up to 24 hours at room temperature (23°C - 27°C) during administration.
Do not take Naglazyme if it contains visible particles.
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 help to protect the environment.
6. Contents of the pack and other information
What Naglazyme contains
- The active substance is galsulfase. One ml of Naglazyme contains 1 mg galsulfase. One vial of 5 ml contains 5 mg galsulfase. Galsulfase is recombinant human N-acetylgalactosamine 4-sulfatase produced by genetically engineered Chinese Hamster Ovary (CHO) cells. - The other ingredients are: sodium chloride, sodium phosphate monobasic, monohydrate, sodium phosphate dibasic, heptahydrate, polysorbate 80, water for injections.
What Naglazyme looks like and contents of the pack
Naglazyme is supplied as a concentrate for solution for infusion. The clear to slightly opalescent and colourless to pale yellow concentrate must be free of visible particles. The solution must be diluted further before it can be infused.
Pack sizes: 1 and 6 vials. Not all package sizes may be marketed.
Marketing Authorization Holder
BioMarin International Limited
Shanbally, Ringaskiddy
County Cork, P43 R298
Ireland
Manufacturer
BioMarin International Limited Shanbally, Ringaskiddy County Cork, P43 R298 Ireland
This leaflet was last revised in 11/2022
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English
1 mg/ml concentrate for solution for infusion
Galsulfase
PACKAGE LEAFLET: INFORMATION FOR THE USER
The following information is intended for medical or healthcare professionals only:
Naglazyme should not be mixed with other medicinal products in the same infusion, except for those mentioned below.
Each vial of Naglazyme is intended for single use only. The concentrate for solution for infusion has to be diluted with sodium chloride 9 mg/ml (0.9%) solution for infusion using aseptic technique. It is recommended that the diluted Naglazyme solution be administered to patients using an infusion set equipped with a 0.2 µm in-line filter.
Any unused product or waste material is to be disposed of in accordance with local requirements.
Preparation of the Naglazyme Infusion (Use Aseptic Technique)
The number of vials to be diluted based on the individual patient’s weight must be determined and removed from the refrigerator approximately 20 minutes in advance in order to allow them to reach room temperature.
Before dilution, each vial is to be inspected for particulate matter and discolouration. The clear to slightly opalescent and colourless to pale yellow solution must be free of visible particles.
A volume of the sodium chloride 9 mg/ml (0.9%) solution for infusion is to be withdrawn and discarded from a 250 ml infusion bag equal to the total volume of Naglazyme to be added. 100 ml infusion bags should be considered for patients who are susceptible to fluid volume overload and weigh less than 20 kg; in this case the infusion rate (ml/min) should be decreased so that the total duration remains no less than 4 hours. When using 100 ml bags, the volume of Naglazyme may be added directly to the infusion bag.
The volume of Naglazyme is to be slowly added to the sodium chloride 9 mg/ml (0.9%) solution for infusion.
The solution is to be mixed gently for infusion.
The solution is to be visually inspected for particulate matter prior to use. Clear and colourless solutions without visible particles should be used.
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