MEPSEVII 2 mg/ml CONCENTRATE FOR INFUSION SOLUTION

Introduction

Package Leaflet: Information for the User

Mepsevii 2mg/ml concentrate for solution for infusion

vestronidase alfa

This medicine 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.
• This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet (see section 4).

Contents of the pack

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

1. What is Mepsevii and what is it used for

What is Mepsevii

Mepsevii contains a recombinant human enzyme called vestronidase alfa. This belongs to a group of medicines called "enzyme replacement therapies". It is given to adults and children of all ages with MPS VII to treat the non-neurological manifestations of the disease (mucopolysaccharidosis VII, also known as Sly syndrome).

What is MPS VII

MPS VII is a genetic inherited disease in which the body does not produce enough of an enzyme called beta-glucuronidase.

• This enzyme breaks down mucopolysaccharides, which are a type of sugar.
• Mucopolysaccharides are made by the body and help form bones, cartilage, skin, and tendons.
• These sugars are constantly being recycled: new ones are created and old ones are broken down.
• If there is not enough beta-glucuronidase, some of these sugars build up in cells, causing damage to the body.

How Mepsevii works

This medicine replaces beta-glucuronidase, helping to break down the sugars that build up in the tissues of people with MPS VII, preventing them from accumulating and causing symptoms of the disease.

• Treatment may improve some signs and symptoms of the disease, such as walking problems and fatigue.

If treatment is started in early childhood, it may slow down the progression of the disease and reduce permanent damage.

2. What you need to know before you receive Mepsevii

Do not receive Mepsevii

• if you are allergic to vestronidase alfa or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor before receiving Mepsevii.

The effects of treatment with vestronidase alfa will be evaluated periodically, and if no clear benefits are seen (including stabilization of clinical manifestations of the disease), discontinuation of treatment will be considered. Stopping treatment may cause significant worsening of the patient's clinical condition.

You should know that administration of vestronidase alfa does not correct irreversible complications of the disease (e.g., bone deformities).

Examine the side effects

• You may experience a reaction to the administration of the drug known as "infusion reaction". This includes any side effect that occurs during infusion or the day after, including an allergic reaction (see section 4 "Possible side effects"). If you experience an infusion reaction, tell your doctor immediately.
• If you experience an allergic reaction during infusion, your doctor may slow down or stop the infusion. Your doctor may also give you other medicines to treat the allergic reaction, such as an antihistamine, a corticosteroid, or an antipyretic (a medicine that lowers fever).

Other symptoms you should examine

• If you experience neck or back pain, numbness in your arms or legs, or loss of control of sphincters, tell your doctor immediately.These symptoms could be signs of a disease caused by spinal cord compression.

Other medicines and Mepsevii

Tell your doctor if you are using, have recently used, or might use any other medicines.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or plan to become pregnant, consult your doctor before using this medicine.

Unless absolutely necessary, Mepsevii will not be given to you if you are pregnant. Discuss with your doctor if the benefits of Mepsevii outweigh the potential risks to the unborn baby, as there is no experience with the use of Mepsevii during pregnancy.

It is not known if Mepsevii passes into breast milk, but it is not expected to pass into your baby. Discuss with your doctor if the benefits of Mepsevii during breastfeeding outweigh the potential risks to the baby.

Driving and using machines

Mepsevii is unlikely to affect your ability to drive or use machines.

Mepsevii contains sodium

This medicine contains 17.8 mg of sodium (the main component of cooking/table salt) per 5 ml vial and is administered with a 9 mg/ml sodium chloride solution as a diluent. Therefore, each vial is equivalent to 1.8% of the maximum recommended daily sodium intake for an adult. Keep this in mind if you are on a low-sodium diet.

3. How Mepsevii is administered

Your doctor will start and monitor treatment with Mepsevii.

• Your doctor or nurse will give you Mepsevii into a vein through infusion (drip).
• This medicine must be diluted before administration.
• Your doctor may also give you other medicines to treat possible allergic reactions, such as an antihistamine, a corticosteroid, or an antipyretic (a medicine that lowers fever).

Dose

The dose you receive will depend on your weight.

• The recommended dose is 4 mg per kilogram of body weight.
• This dose is given every two weeks through intravenous infusion.
• Each infusion will be given over about 4 hours.

If you receive more Mepsevii than you should

Administration of Mepsevii will be supervised and monitored by your doctor so that the dose is correct and measures can be taken if necessary.

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 receiving the medicine or during the day after (reactions associated with infusion).

If you experience any reaction like this, contact your doctor immediatelyas you may need urgent treatment.

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

• Severe allergic reaction (anaphylactoid reaction) – Symptoms of a severe allergic reaction include, among others, shortness of breath, wheezing, difficulty breathing, and swelling of the face and tongue. Your doctor may give you other medicines to treat the allergic reaction, such as an antihistamine, a corticosteroid, or an antipyretic (a medicine that lowers fever).
• Hives
• Rash
• Swelling at the infusion site, including fluid leaking into the tissues surrounding the vein (infusion site swelling or extravasation).

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

• Itching of the skin (pruritus)
• Loose stools (diarrhea)
• Fever with involuntary muscle contractions of the face or limbs (febrile convulsion)

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 via 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 Mepsevii

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date ("EXP") stated on the carton. 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.
• Store in the original package to protect from light.
• Do not use this medicine if you notice particles in it.

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 further information

What Mepsevii contains

• The active substance is vestronidase alfa. Each ml of concentrate contains 2 mg of vestronidase alfa. Each 5 ml vial of concentrate contains 10 mg of vestronidase alfa.
• The other ingredients are: sodium dihydrogen phosphate dihydrate, sodium chloride, histidine, polysorbate 20, and water for injections (regarding sodium, see section 2 "Mepsevii contains sodium").

Appearance of Mepsevii and pack contents

Mepsevii is provided as a concentrate for solution for infusion (sterile concentrate). The concentrate is colorless to slightly yellow and should not contain visible particles. It is provided in a transparent glass vial with a rubber stopper and an aluminum seal with a plastic cap.

Pack size: 1 vial of 5 ml

Marketing authorisation holder

Ultragenyx Germany GmbH

Rahel-Hirsch-Str. 10

10557 Berlin

Germany

Manufacturer

Ultragenyx Netherlands B. V.

Evert van de Beekstraat 1, Unit 104

1118 CL Schiphol

Netherlands

Millmount Healthcare Ltd.

Block-7, City North Business Campus

Stamullen, Co. Meath,

Ireland

You can request more information about this medicine from the local representative of the marketing authorisation holder:

BE, BG, CZ, DK, DE, EE, IE, EL, ES, HR, IT, CY, LV, LT, LU, HU, MT, NL, AT, PL, PT, RO, SI, SK, FI, SE, XI

Ultragenyx Germany GmbH, DE

Tel: + 49 30 20179810

FR

Ultragenyx France SAS, FR

Tel: + 33 185 653761 or 0800 91 79 24 (green number)

Date of last revision of this leaflet: MM/AAAA.

This medicine has been authorised under "exceptional circumstances". This means that due to the low prevalence of this disease, it has not been possible to obtain complete information on this medicine.

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

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency website: http://www.ema.europa.eu. There are also links to other websites on rare diseases and orphan medicines.

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