Mepsevii

Patient Information Leaflet

Mepsevii 2 mg/mL concentrate for solution for infusion

vestronidase alfa
This medicinal product is under additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you experience while taking this medicine. See the end of section 4 for information on how to report side effects.
Please read all of this leaflet carefully before taking this medicine, as it contains important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor.
• This medicine has been prescribed for you only. Do not give it to others, even if their symptoms are the same as yours, as it may be harmful.
• If you experience any side effect, including those not listed in this leaflet, tell your doctor. This includes any possible side effect not listed in this patient information leaflet (see section 4).

Contents of this leaflet

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

1. What Mepsevii is and what it is used for

What Mepsevii is
Mepsevii contains an enzyme called vestronidase alfa. It belongs to a group of medicines known as enzyme replacement therapies. It is used in adults and children of all ages who have MPS VII to treat non-neurological manifestations of the disease (mucopolysaccharidosis type VII, also known as Sly syndrome).

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

• This enzyme helps break down sugars in the body called mucopolysaccharides.
• Mucopolysaccharides are produced in the body and help develop bones, cartilage, skin, and tendons.
• These sugars are continuously recycled – new ones are produced and old ones are broken down.
• Without sufficient beta-glucuronidase, some of these sugars accumulate in cells, causing damage to the body.

How Mepsevii works
This medicine replaces beta-glucuronidase – thereby helping to break down the sugars that accumulate in the tissues of patients affected by MPS VII.

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

Initiating early treatment in children may halt disease progression and reduce permanent damage.

2. What you should know before taking Mepsevii

Do not take Mepsevii

• if you have had a severe allergic reaction to vestronidase alfa or to any of the other excipients of this medicine (listed in section 6).

Warnings and precautions
Talk to your doctor before taking Mepsevii.
The effects of treatment with vestronidase alfa should be evaluated periodically, and discontinuation
of treatment should be considered if no clear benefits are observed (including stabilization of disease manifestations). Discontinuation of treatment
may lead to a significant worsening of the patient's clinical condition.
It should be noted that administration of vestronidase alfa does not affect irreversible complications (e.g., skeletal system deformities).
Be aware of possible side effects during or shortly after infusion of Mepsevii.

• You may experience side effects while receiving Mepsevii or up to one day afterwards. These side effects are known as infusion reactions because they are caused by the infusion (intravenous drip) of the medicine. They may include an allergic reaction (see section 4). If you experience an infusion reaction, inform your doctor immediately.
• If you experience an allergic reaction during the infusion, your doctor may slow down or stop the infusion. Your doctor may give you (or may have already given you) other medicines to manage allergic reactions, such as antihistamines, corticosteroids, or an antipyretic (a medicine to reduce fever). Other symptoms to watch for:
• If you experience neck or back pain, numbness in your arms or legs, or if you are unable to control your urine or bowel movements, inform your doctor immediately. These problems may be signs of the disease and could be caused by pressure on the spinal cord.

Other medicines and Mepsevii
Ask your doctor if you are currently taking, have recently taken, or might take other medicines.
Pregnancy and breastfeeding
If you are pregnant, think you might be pregnant, are planning a pregnancy, or are breastfeeding,
please consult your doctor before taking this medicine.
Do not take Mepsevii during pregnancy unless treatment is strictly necessary.
Consult your doctor to determine whether the benefits of using Mepsevii outweigh the potential
risks to the unborn child. This is because there are no data available on the use of Mepsevii during pregnancy.
It is not known whether Mepsevii passes into breast milk, but it is not expected that the medicine will pass to the infant. Consult your doctor to determine whether the benefits of using Mepsevii outweigh the potential risks to the infant during breastfeeding.
Driving and using machines
The effects of Mepsevii on the ability to drive vehicles or operate machinery are unknown.
Mepsevii contains sodium
This medicine contains 17.8 mg of sodium (the main component of table salt) in each 5 mL vial and is administered with sodium chloride at a concentration of 9 mg/mL as diluent. Each vial administered therefore corresponds to 1.8% of the maximum daily recommended sodium intake for an adult. Take this into account if you are on a low-sodium diet.

3. How to take Mepsevii

Treatment with Mepsevii must be initiated and monitored by a doctor.

• Mepsevii will be administered to you by a doctor or nurse as an intravenous infusion (intravenous drip).
• The medicine must be diluted before administration.
• Your doctor may give you (or may have already given you) certain medicines to manage allergic reactions, such as antihistamines, corticosteroids, or an antipyretic (a medicine to reduce fever).

Dosage
The dose you will receive depends on your body weight.

• The recommended dose is 4 mg per kilogram of body weight.
• The dose is administered every two weeks via an intravenous drip (intravenous infusion).
• Each infusion will last approximately 4 hours.

If you receive more Mepsevii than you should
Mepsevii is administered and monitored by your doctor. He or she will ensure that you receive the correct dose and will take appropriate action if necessary.
If you have any further questions about the use of this medicine, consult your doctor.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
Side effects have occurred mainly while patients were receiving the medicine or within one day after the infusion (infusion reactions).

Serious side effects
Severe allergic reaction (very common: may affect more than 1 in 10 people):
Tell your doctor or nurse immediately if you experience any of the following symptoms of a severe allergic reaction (anaphylactoid reaction). The infusion will be stopped immediately, and your doctor may give you (or may have already given you) other medicines to manage the allergic reaction, such as an antihistamine, a corticosteroid, or an antipyretic (a medicine to reduce fever).
Symptoms of a severe allergic reaction may include shortness of breath, wheezing, difficulty breathing, and swelling of the face and tongue.

Other side effects
Tell your doctor immediately if you notice any of the following side effects; you may need urgent medical treatment:

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

• Hives (urticaria)
• Rash
• Swelling at the infusion site, including leakage into the surrounding tissue (infusion site swelling or infusion site extravasation)

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

• Itching of the skin
• Loss of bowel control (diarrhea)
• Fever with involuntary muscle contractions of the face or limbs (febrile convulsions)
• Swelling around the infusion site

Reporting of side effects
If you get any side effect, including ones not listed in this leaflet, talk to your doctor. You can also report side effects directly via the national reporting system listed in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store Mepsevii

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the carton after the word
"EXPIRY". The expiry date refers to the last day of that month.
Unopened vials:

• Store in a refrigerator (from 2 °C to 8 °C).
• Do not freeze.
• Keep in the original packaging to protect the medicine from light.
• Do not use this medicine if you notice any particles.

Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

6. Package contents and other information

What Mepsevii contains

• The active substance is vestronidase alfa. Each mL of concentrate contains 2 mg of vestronidase alfa. Each 5 mL concentrate vial contains 10 mg of vestronidase alfa.
• The other excipients are: disodium hydrogen phosphate dihydrate, sodium chloride, histidine, polysorbate 20, and water for injections (for sodium content, see section 2 under "Mepsevii contains sodium").

Description of the appearance of Mepsevii and contents of the pack
Mepsevii is supplied as a concentrate for solution for infusion (sterile concentrate). The concentrate is colourless to slightly yellow and should be free from visible particles. It is supplied in a clear glass vial with a rubber stopper, an aluminium seal, and 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
The Netherlands
For further information about this medicinal product, please contact 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, UK(NI)
Ultragenyx Germany GmbH, DE
Tel/Tél/Teл./Tlf/Τηλ/Puh/Sími: + 49 30 20179810
FR
Ultragenyx France SAS, FR
Tél: + 33 1 85 65 37 61 or 0800 91 79 24 (toll-free number)
This medicinal product has been granted a marketing authorisation under "exceptional circumstances". This means that, due to the rarity of the disease, it has not been possible to obtain complete information about this medicinal product. The European Medicines Agency will review any new information on this medicinal product annually, and this patient information leaflet will be updated if necessary.
Other sources of information
More detailed information on this medicinal product is available on the website of the European Medicines Agency: https://www.ema.europa.eu. In addition, links to other websites on rare diseases and their therapeutic treatments are provided.