Prospect: information for the user

Mepsevii 2mg/ml concentrate for solution for infusion

vestronidasa alfa

This medicine is subject to additional monitoring, which will speed up 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 prospect carefully before starting to use this medicine, because it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you have any other questions, consult your doctor.
• This medicine has been prescribed only for you, and you must not give it to other people, even if they have the same symptoms as you, as it may harm them.
• If you experience adverse effects, consult your doctor, even if they are not listed in this prospect (see section 4).

1.What is Mepsevii and what it is used for

2.What you need to know before starting to receive Mepsevii

3.How to administer Mepsevii

4.Possible adverse effects

5.Storage of Mepsevii

6.Contents of the package and additional information

What is Mepsevii

Mepsevii contains a human recombinant enzyme called vestronidase alfa. This belongs to a group of medications called "enzyme replacement therapies". It is administered 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 hereditary disease in which the body does not produce a sufficient amount of an enzyme known as beta-glucuronidase.

• This enzyme breaks down mucopolysaccharides, which are a type of sugar.
• Mucopolysaccharides are produced by the body and help form bones, cartilages, skin, and tendons.
• These sugars are constantly recycled: new ones are created and old ones are destroyed.
• If there is not enough beta-glucuronidase, part of these sugars is deposited in cells causing damage to the body.

How Mepsevii works

This medication replaces beta-glucuronidase, facilitating the breakdown of sugars that are deposited in the tissues of people with MPS VII, preventing them from accumulating and causing the symptoms of the disease

• The treatment may improve some signs and symptoms of the disease, such as gait problems and fatigue.

If treatment is initiated at an early stage of childhood, the progression of the disease can be slowed and permanent damage can be reduced.

Do not receive Mepsevii

-if you are allergic to vestronidase alfa or any of the other components of this medication (listed in section 6).

Warnings and precautions

Consult your doctor before receiving Mepsevii.

The effects of treatment with vestronidase alfa will be evaluated periodically, and if no clear beneficial effects (including stabilization of clinical manifestations of the disease) are observed, the definitive suspension of treatment will be assessed. Suspension of treatment may cause a significant deterioration of the patient's clinical condition.

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

Examine adverse effects

• You may experience a reaction to the administration of the drug known as "infusion reaction"). This includes any adverse effect that occurs during infusion or the following day, including an allergic reaction (see section 4 "Possible adverse effects"). If you experience an infusion reaction,inform your doctor immediately.
• If you experience an allergic reaction during infusion, the doctor may slow down or stop the infusion. It is possible that the doctor has also administered (or will administer) other medications to treat the allergic reaction, such as an antihistamine, corticosteroid, or antipyretic (a medication that lowers fever).

Other symptoms to examine

• If you experience neck or back pain, numbness in the arms or legs, or loss of bladder control,inform your doctor immediatelyThese symptoms may be signs of a spinal cord compression disease

Other medications and Mepsevii

Inform your doctor if you are using, have used recently, or may need to use any other medication.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, or think you may be pregnant, consult your doctor before using this medication.

Unless absolutely necessary, Mepsevii will not be administered to you if you are pregnant. Discuss with your doctor if the benefits of Mepsevii outweigh the possible risks for the fetus, as there is no experience with the use of Mepsevii during pregnancy.

The passage of Mepsevii into breast milk is unknown, but it is not expected to pass to your baby. Discuss with your doctor if the benefits of Mepsevii during breastfeeding outweigh the possible risks for the baby.

Driving and operating machines

Mepsevii is unlikely to affect driving or operating machines.

Mepsevii contains sodium

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

The doctor will initiate and monitor treatment with Mepsevii.

• The doctor or nursing staff will administer Mepsevii through a vein via infusion (drip).
• This medication must be diluted before administration.
• The doctor may administer (or have administered) medication to treat possible allergic reactions, such as an antihistamine, corticosteroid, or antipyretic (a medication 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 administered every two weeks via intravenous infusion (venous drip).
• Each infusion will be administered over a period of 4 hours.

If you receive more Mepsevii than you should

The administration of Mepsevii will be supervised and monitored by your doctor to ensure the correct dose and to take measures if necessary.

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

Like all medicines, this medicine can produce adverse effects, although not all people will experience them.

Adverse effects were mainly observed while the patients were receiving the medicine or over the course of the following day (reactions associated with infusion).

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

Adverse effectsvery common(affect more than 1 in 10people):

• Severe allergic reaction (anaphylactoid reaction) – The symptoms of a severe allergic reaction include, among others, shortness of breath, wheezing (respiratory wheezes), difficulty breathing, and swelling of the face and tongue. It is possible that your doctor will administer (or has administered) other medicines to treat the allergic reaction, such as an antihistamine, a corticosteroid, or an antipyretic (a medicine that lowers fever).
• Urticaria
• Rash
• Swelling at the infusion site, including the leakage of liquid into the tissues surrounding the vein (swelling at the infusion site or extravasation at the infusion site).

Adverse effectscommon(may affect up to 1 in 10people):

• Itching on the skin (pruritus)
• Loose stools (diarrhea)
• Fever with involuntary contractions of the facial or limb muscles (febrile convulsion)

Reporting Adverse Effects

If you experience adverse effects, consult your doctor, even if they are possible adverse effects that do not appear in this leaflet. You can also report them directly through thenational notification system included in theAppendix V. By reporting adverse effects, you can contribute to providing more information about the safety of this medicine.

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

Do not use this medication after the expiration date ("CAD") that appears on the box. The expiration date is the last day of the month indicated.

Unopened vials:

• Store in refrigerator (between 2°C and 8°C).
• Do not freeze.
• Store in the original container to protect it from light.
• Do not use this medication if you observe particles inside.

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

What Mepsevii contains

• The active ingredient is called vestronidase alfa. Each milliliter of concentrate contains 2 mg of vestronidase alfa. Each vial of 5 ml of concentrate contains 10 mg of vestronidase alfa.
• The other ingredients are: dihydrogen phosphate of sodium dihydrate, sodium chloride, histidine, polisorbate 20 and water for injection (as for sodium, see section 2 "Mepsevii contains sodium").

Appearance of Mepsevii and contents of the pack

Mepsevii is supplied as a concentrate for solution for infusion (sterile concentrate). The concentrate, between colourless and slightly yellow, must not contain visible particles. It is supplied in a transparent glass vial with a rubber stopper and an aluminium seal with a plastic capsule.

Size of the pack: 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 by contacting 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

Phone/Phone/Te?./Tlf/Τηλ/Puh/Sími: + 49 30 20179810

FR

Ultragenyx France SAS, FR

Phone: + 33 185 653761 or 0800 91 79 24 (free phone number)

Revision date of this leaflet: MM/YYYY.

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

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

Other sources of information

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

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