PROSPECTO: INFORMACIÓN FORTHEUSER

Naglazyme 1 mg/ml concentrate for infusion solution

Galsulfase

This medicine is subject to additional monitoring, which will expedite the detection of new information about its safety. You can contribute by reporting any adverse effects you may experience. The final part of section 4 includes information on how to report these adverse effects.

Read this prospectus carefully before starting to use the medicine, as it contains important information for you.

• Keep this prospectus, as you may need to refer to it again.
• If you have any doubts, consult your doctor.
• If you consider any of the adverse effects you are experiencing to be severe or if you notice any adverse effect not mentioned in this prospectus, inform your doctor or pharmacist.

1.What is Naglazyme and what it is used for

2.What you need to know before starting to use Naglazyme

3.How to use Naglazyme

4.Possible adverse effects

5.Storage of Naglazyme

6.Contents of the package and additional information

Naglazyme is used to treat patients with MPS VI (Mucopolisacaridosis VI).

Individuals affected by MPS VI have a low or absent level of an enzyme called N-acetylgalactosamine 4-sulfatase that breaks down specific substances (glycosaminoglycans) in the body. As a result, these substances are not 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 it worksNaglazyme

This medication contains a recombinant enzyme called galsulfase that can replace the missing natural enzyme in patients with MPS VI. It has been shown that treatment improves walking endurance and reduces the concentrations of glycosaminoglycans in the body. This medication may improve the symptoms of MPS VI.

No useNaglazyme

-If you have experienced severe or potentially life-threatening allergic reactions (hypersensitivity) to galsulfase or any of the other components of Naglazyme and re-administration of the medication was unsuccessful.

Warnings and precautions

-If you are being treated with Naglazyme, you may develop infusion-related reactions. An infusion-related reaction is any adverse effect that appears during the infusion or up to the end of the infusion day (see section 4 “Possible adverse effects”). If you experience such a reaction,you must immediately contact your doctor.

-If you experience an allergic reaction, your doctor may slow down or stop your infusion. Your doctor may also give you additional medications to treat any allergic reaction.

-If you have a fever or difficulty breathing before using this medication, consult with your doctor about the possibility of delaying the Naglazyme infusion.

-This medication has not been studied in patients with liver or kidney problems. Consult your doctor if you have renal or hepatic insufficiency.

-Talk to your doctor if you experience muscle pain, numbness in the arms or legs, or any gastrointestinal or urinary problems, as these may be caused by spinal cord compression.

Other medications and Naglazyme

Inform your doctor if you are using or have recently used other medications, including those obtained without a prescription.

Pregnancy and breastfeeding

Naglazyme should not be administered during pregnancy, unless clearly necessary. Consult your doctor or pharmacist before using any medication. It is unknown whether galsulfase is excreted in breast milk, so you should interrupt breastfeeding during treatment with Naglazyme. Consult your doctor or pharmacist before using any medication.

Driving and operating machinery

No studies have been conducted on the effect on the ability to drive and operate machinery.

This medication contains sodium

This medication contains 0.8 mmol (18.4 mg) of sodium (main component of table salt/for cooking) in each 5 ml vial. This is equivalent to % of the recommended daily maximum sodium intake for an adult and is administered in a 9 mg/ml sodium chloride solution...

Your doctor or nurse will administer Naglazyme.

The dose you receive will depend on your body weight. The recommended dose is 1mg/kg of body weight, administered once a week by slow infusion into a vein (intravenous infusion). Each infusion will last approximately 4 hours. During the first hour, the infusion rate will be slow (approximately 2.5% of the total solution) and the remaining volume (approximately 97.5%) will be administered during the next 3 hours.

If you are given more Naglazyme than you should

Naglazyme is administered under the supervision of a nurse or doctor, who will ensure that the dose you receive is correct and take action if necessary.

If you forgot that Naglazyme was administeredNaglazyme

If you have missed a Naglazyme infusion, contact your doctor.

If you have any other questions about the use of this medication, ask your doctor.

Like all medicines, this medicine can cause side effects, although not everyone will experience them.

Adverse effects were mainly observed while patients were receiving the medicine or shortly after (“perfusion-related reactions”) . The most serious adverse effects were facial inflammation and fever (very common), longer than normal spaces between breaths, , respiratory difficulty, , asthma, and urticaria (common); and tongue and throat inflammation, and severe allergic reaction to this medicine (with an unknown frequency).

If you experience any of these reactions,inform your doctor immediately.You may need to receive additional medications to prevent an allergic reaction (e.g. antihistamines or corticosteroids) or to reduce fever (antipyretics).

The most common symptoms of perfusion-related reactions include fever, chills, skin rash, urticaria, and difficulty breathing.

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

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

Other adverse effects with an unknown frequency :

If you experience any of these symptoms, or other symptoms not listed in this leaflet, consult your doctor immediately.You can also report them directly through thenational reporting system included in theAppendix V. By reporting adverse effects, you can contribute to providing more information on the safety of this medicine.

Keep out of the sight and reach of children.

Do not use this medication after the expiration date that appears in the vial after EXP. The expiration date is the last day of the month indicated.

Unopened vials:

Store in the refrigerator (between 2 °C and 8 °C).

Do not freeze.

Diluted solutions: Chemical and physical stability has been demonstrated for use up to 4 days at room temperature (23 °C - 27 °C).

To avoid microbial contamination, the product must be used immediately. If not used immediately, the storage times and conditions in use are the responsibility of the user, and normally should not exceed 24 hours at 2 °C - 8 °C, followed by up to 24 hours at room temperature (23 °C - 27 °C) during administration.

Do not use Naglazyme if you observe visible particles.

Medicines should not be disposed of through the drains or in the trash. Ask your pharmacist how to dispose of the containers and medicines that you no longer need. This will help protect the environment.

Composition of Naglazyme

-The active ingredient is galsulfase. Each milliliter of Naglazyme contains 1 mg of galsulfase. A vial of 5 ml contains 5 mg of galsulfase. Galsulfase is human recombinant N-acetylgalactosamine 4-sulfatase produced in genetically modified Chinese hamster ovary (CHO) cells.

-The other components are: sodium chloride, monobasic sodium phosphate monohydrate, dibasic sodium phosphate heptahydrate, polisorbate 80, water for injection.

Appearance of the product and contents of the pack

Naglazyme is supplied as a concentrate for solution for infusion. The concentrate varies from transparent to slightly opalescent, and from colorless to pale yellow, and should not contain visible particles. The solution must be diluted before it can be infused.

Pack sizes:1 and 6 vials. Some pack sizes may only be marketed.

Last review date of this leaflet: MM/YYYY

This medicine has been authorized under "exceptional circumstances".

This type of authorization means that due to the rarity of your disease, it has not been possible to obtain a complete information about this medicine.

The European Medicines Agency will review annually any new information about the medicine that may become available and this leaflet will be updated as necessary.

Further information on this medicine is available on the website ofthe European Medicines Agencyathttp://www.ema.europa.eu/. It also provides links to other websites about rare diseases and orphan medicines.

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This information is intended only for healthcare professionals:

Naglazyme should not be mixed with any other medicine in the same infusion, except those mentioned below.

Each vial of Naglazyme is for single use. The concentrate for solution for infusion must be diluted with a sodium chloride 9mg/ml (0.9%) infusion solution using aseptic technique. It is recommended that the diluted Naglazyme solution be administered to patients using an infusion device equipped with an in-line filter of 0.2µm.

Unused medicine and all materials that have been in contact with it will be disposed of in accordance with local regulations.

Preparation of the Naglazyme infusion (use aseptic technique)

The number of vials to be diluted will be determined according to the patient's weight and will be removed from the refrigerator approximately 20 minutes in advance to allow them to reach room temperature.

Before dilution, each vial will be visually inspected for the presence of particles or color changes. The solution, which varies from transparent to slightly opalescent and from colorless to pale yellow, should be free of visible particles.

A volume of sodium chloride 9mg/ml (0.9%) infusion solution equal to the total volume of Naglazyme to be added will be withdrawn and discarded from a 250ml infusion bag. The use of 100ml infusion bags may be considered in patients sensitive to fluid overload and weighing less than20kg; in such cases, the infusion rate (ml/min) will be reduced so that the total duration remains not less than 4 hours. If 100ml infusion bags are used, the Naglazyme volume may be added directly to the infusion bag.

The Naglazyme volume will be added slowly to the sodium chloride 9mg/ml (0.9%) infusion solution.

The solution will be mixed gently for infusion.

The solution will be visually inspected for particles before use. Only transparent and colorless solutions without visible particles will be used.