NAGLAZYME 1 mg/ml CONCENTRATE FOR INFUSION SOLUTION

Introduction

PROSPECT: INFORMATION FORTHEUSER

Naglazyme 1 mg/ml concentrate for solution for infusion

Galsulfase

This medicinal product is subject to additional monitoring, which will allow for quick identification of new safety information. You can help by reporting any side effects you may get. The last section of this leaflet includes information on how to report side effects.

Read all of this leaflet carefully before you start using this medicine because it contains important information for you.

• Keep this leaflet, you may need to read it again.
• If you have any further questions, ask your doctor or pharmacist.
• If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet.

Contents of the pack:

1. What is Naglazyme and what is it used for
2. What you need to know before you use Naglazyme
3. How to use Naglazyme
4. Possible side effects
5. Storage of Naglazyme
6. Contents of the pack and other information

1. What is Naglazyme and what is it used for

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

People with MPS VI have low or no levels 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 be. They accumulate in many tissues in the body, causing the symptoms of MPS VI.

HowNaglazyme works

This medicine contains a recombinant enzyme called galsulfase that can replace the natural enzyme that is missing in patients with MPS VI. It has been shown that treatment improves walking and stair-climbing ability, and reduces the levels of glycosaminoglycans in the body. This medicine may improve the symptoms of MPS VI.

2. What you need to know before you use Naglazyme

Do not useNaglazyme

• If you have experienced severe or life-threatening allergic reactions (hypersensitivity) to galsulfase or any of the other components of Naglazyme and readministration of the medicine was not successful.

Warnings and precautions

• If you are treated with Naglazyme, you may develop infusion-related reactions. An infusion-related reaction is any adverse event that occurs during the infusion or up to the end of the infusion day (see section 4 “Possible side effects”). If you experience such a reaction, you should contact your doctor immediately.
• If you experience an allergic reaction, your doctor may slow down or stop your infusion. Your doctor may also give you additional medicines to treat any allergic reaction.
• If you have a fever or difficulty breathing before using this medicine, consult your doctor about the possibility of delaying the infusion of Naglazyme.
  • If you have underlying heart disease, inform your doctor at any time while receiving treatment with Naglazyme. Your doctor may adjust the infusion based on this information.

• This medicine has not been studied in patients with liver or kidney problems. Consult your doctor if you have renal or hepatic impairment.
• Talk to your doctor if you experience muscle pain, numbness in your arms or legs, or any intestinal or urinary problems, as they may be caused by pressure on the spinal cord.

Other medicines and Naglazyme

Tell your doctor if you are using or have recently used other medicines, 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 medicine. It is not known whether galsulfase is excreted in breast milk, so breastfeeding should be discontinued during treatment with Naglazyme. Consult your doctor or pharmacist before using any medicine.

Driving and using machines

No studies have been performed on the effects on the ability to drive and use machines.

This medicine contains sodium

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

3. How to use Naglazyme

Your doctor or nurse will administer Naglazyme to you.

The dose you receive will depend on your body weight. The recommended dose is 1 mg/kg body weight, administered once a week by slow infusion into a vein (as an intravenous infusion). Each infusion will take 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 over the next 3 hours.

If you are given too much Naglazyme

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

If you miss an infusion ofNaglazyme

If you have missed an infusion of Naglazyme, contact your doctor.

If you have any other questions about 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 receiving the medicine or soon after (“infusion-related reactions”). The most serious side effects were facial swelling and fever (very common), prolonged intervals between breaths, difficulty breathing, asthma, and hives (common); and swelling of the tongue and throat, and severe allergic reaction to this medicine (with unknown frequency).

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

The most common symptoms of infusion-related reactions include fever, chills, rash, hives, and difficulty breathing.

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

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

Other side effects with unknown frequency:

If you experience any of these symptoms, or any other symptoms that do not appear in this leaflet, tell your doctor immediately. You can also report side effects directly through the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Naglazyme

Keep out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the vial after EXP. The expiry date is the last day of the month stated.

Unopened vials:

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

Do not freeze.

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

To avoid microbial contamination, the product should be used immediately. If not used immediately, the in-use storage times and conditions 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 notice visible particles.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. This will help protect the environment.

6. Contents of the pack and other information

Composition of Naglazyme

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

• The other ingredients are: sodium chloride, sodium phosphate monobasic monohydrate, disodium phosphate heptahydrate, polysorbate 80, water for injections.

Appearance and pack contents

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. Not all pack sizes may be marketed.

Date of last revision of this leaflet: MM/YYYY

This medicine has been authorised under “exceptional circumstances”.

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

The European Medicines Agency will review any new information that becomes available every year and this leaflet will be updated as necessary.

Detailed information on this medicine is available on the website of the European Medicines Agency http://www.ema.europa.eu/. It also provides links to other websites on 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 for those mentioned below.

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

Disposal of unused medicine and all materials that have come into contact with it should be done in accordance with local regulations.

Preparation of Naglazyme infusion (using aseptic technique)

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

Prior to 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 9 mg/ml (0.9%) solution for infusion will be withdrawn from a 250 ml infusion bag and discarded, equal to the total volume of Naglazyme to be added. The use of 100 ml infusion bags may be considered in patients sensitive to fluid volume overload and weighing less than 20 kg; in such cases, the infusion rate (ml/min) will be reduced so that the total duration is not less than 4 hours. If 100 ml bags are used, the volume of Naglazyme may be added directly to the infusion bag.

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

The solution will be gently mixed for infusion.

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