1 mg / ml concentrate for solution for infusion
Galsulfase
What this medicine is and what it is used for
Naglazyme is used to treat patients with MPS VI (mucopolysaccharidosis VI).
People with MPS VI have either very little or nothing at all of an enzyme called N-acetylgalactosamine-4-sulfatase, which is needed to break down certain special substances (glycosaminoglycans) in the body. The result is that these substances are not broken down and processed by the body as they should. They accumulate in many of the body’s tissues and give rise to the symptoms of MPS VI.
How this medicine works
This medicine contains a recombinant enzyme called galsulfase. It can replace the natural enzyme that is missing in patients who have MPS VI. The treatment has been shown to improve the ability to walk and walk upstairs and reduce the levels of glycosaminoglycans in the body. This medicine may improve the symptoms of MPS VI.
What you need to know before you are given this medicine
You must not be given this medicine
- If you have had severe or life-threatening allergic reactions ( hypersensitivity reactions ) to bile sulphase or to any of the other ingredients of Naglazyme and repeated treatment with the medicine did not work well.
Warnings and cautions
- If you are being treated with Naglazyme, you may experience infusion-related reactions. An infusion-related reaction is any type of side effect that occurs during the infusion or during the rest of the day you receive the infusion (see section 4 “Possible side effects”). If you get such a reaction , contact your doctor immediately .
- If you get an allergic reaction , your doctor may slow down or stop the infusion . Your doctor may also give you other medicines to limit the allergic reaction.
- If you get a fever or have difficulty breathing before taking this medicine, talk to your doctor about delaying a Naglazyme infusion .
- This medicine has not been tested in patients with kidney or liver problems. Talk to your doctor if you have impaired kidney or liver function.
- Talk to your doctor if you get muscle pain, numbness in the arms or legs, or have problems with the bowel or bladder, as this can be caused by pressure on the spinal cord.
Other medicines and Naglazyme
Tell your doctor if you are taking or have recently taken any other medicines, including medicines obtained without a prescription.
Pregnancy and breastfeeding
Naglazyme should not be given during pregnancy unless necessary. Ask your doctor or pharmacist for advice before taking any medicine. It is not known whether bile sulfate passes into breast milk. Therefore, you should not breastfeed while you are being treated with Naglazyme. Ask your doctor or pharmacist for advice before taking any medicine.
Driving and using machines
No studies on the effects of Naglazymes on the ability to drive and use machines have been performed.
This medicine contains sodium
Each vial contains 0.8 mmol (18.4 mg) sodium . This should be considered by patients on a low-salt diet.
How to get this medicine
Your doctor or nurse will give you Naglazyme.
The dose you receive is based on your body weight. The recommended dose is 1 mg/kg body weight given weekly by a drip into a blood vessel (an intravenous infusion ). Each infusion takes approximately 4 hours. During the first hour, the infusion will be slow (about 2.5% of the total solution) and the remaining volume (about 97.5%) will be given over the next three hours.
If you take more Naglazyme than you should
Naglazyme is given under the supervision of a nurse or doctor, and he or she will check that you have received the correct dose and, if necessary, take appropriate action.
If you forget to take this medicine
Contact your doctor if you have missed a Naglazyme infusion.
If you have any further questions on the use of this product, ask your doctor.
Possible side effects
Like all medicines, this medicine can cause side effects, although not everybody gets them.
Side effects were mainly seen during or shortly after the patients received the medicine (“infusion-related reactions”). The most serious side effects were swollen face and fever (very common); longer than normal intervals between breathing, difficulty breathing, asthma and hives (common); swollen tongue and throat, and severe allergic reaction to this drug (no known frequency).
Should you experience such a reaction, tell your doctor immediately. You may need to take additional medicines to prevent an allergic reaction (eg antihistamines and/or corticosteroids ) or to lower your fever (antipyretics).
The most common symptoms of infusion-related reactions include fever, chills, rash, hives, and shortness of breath.
Very common side effects (they may affect more than 1 in 10 people):
| Sore throatGastrointestinal inflammation bad reflexesHeadacheEye inflammationBlurred eyePoor hearingHigh blood pressure | Nasal congestionBulging navelVomitingNauseaItchingPain (including ear, abdomen, joint, chest pain)Feeling sick |
Common side effects (these may affect up to 1 in 10 people):
| ShakingLow blood pressure cough | WheezingReddening of the skin |
Other side effects with unknown frequency:
| ShockKnittingDecreased heart rate increased heart rate | Bluish skinPale skinLow oxygen content in the blood paid to breathe |
How to store this medicine
Keep out of sight and reach of children.
Do not use this medicine after the expiry date which is stated on the bottle after EXP. The expiration date is the last day of the specified 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 when used for up to 4 days at room temperature (23 ° C – 27 ° C).
For the product to be safe from a microbiological point of view, it must be used immediately. If the solution is not used immediately, the user is responsible for the storage time and conditions of use. The storage time should not normally exceed 24 hours at 2 ° C – 8 ºC followed by a maximum of 24 hours at room temperature (23 ° C – 27 ° C) during infusion.
Naglazyme should not be used if it contains any visible particles.
The medicine 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.
Contents of the pack and other information
Content declaration
- The active substance is bile sulphase. One ml of Naglazyme contains 1 mg of galsulfase. One 5 ml vial contains 5 mg of bile sulphase. Bile sulfase consists of the human form of the enzyme N-acetylgalactosamine-4-sulfatase. It is manufactured with so-called recombinant technology from genetically modified Chinese hamster ovary cells (CHO cells).
- The other ingredients are: sodium chloride, sodium dihydrogen phosphate monohydrate,disodium hydrogen phosphate heptahydrate, polysorbate 80, water for injections.
What the medicine looks like and contents of the pack
Naglazyme is supplied as a concentrate for infusion solutions (“drip”). The concentrate should be completely clear or slightly opalescent. It should also be colorless to pale yellow and there must be no visible particles in it. The solution must be diluted before it can be used for infusion.
Pack sizes: 1 and 6 vials. Not all pack sizes may be marketed.
Marketing Authorization Holder and Manufacturer
| Marketing Authorisation HolderBioMarin International Limited Shanbally, RingaskiddyCounty Cork, P43 R298Ireland | ManufacturerBioMarin International Limited Shanbally, Ringaskiddy County Cork, P43 R298Ireland |