Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - xenpozyme

Document Subject

Generated Narrative: MedicinalProductDefinition mp1c8a533780954e330a4c10339c9c24f7

identifier: http://ema.europa.eu/identifier/EU/1/22/1659/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Xenpozyme 4 mg powder for concentrate for solution for infusion

type: Full name

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part: nan

type: Invented name part

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part: nan

type: Scientific name part

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type: Strength part

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type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-1c8a533780954e330a4c10339c9c24f7

identifier: http://ema.europa.eu/identifier/EU/1/22/1659/001

status: Final

type: Package Leaflet

category: Raw

date: 2022-02-16 13:28:17+0000

author: Organization ACME industry

title: TEST PURPOSES ONLY - xenpozyme

Attesters

What is in this leaflet

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

What Xenpozyme is Xenpozyme contains an enzyme called olipudase alfa.

What Xenpozyme is used for Xenpozyme is used to treat an inherited disorder called acid sphingomyelinase deficiency (ASMD). It is used in children and adults with ASMD types A/B or B to treat the signs and symptoms of ASMD not related to the brain.

How Xenpozyme works Patients with ASMD lack a properly working version of the enzyme acid sphingomyelinase. This results in build-up of a substance called sphingomyelin, which damages organs such as spleen, liver, heart, lungs and blood. Olipudase alfa acts in the same way as the natural enzyme would, and so acts as a replacement, reducing the build-up of sphingomyelin in the organs and treating the signs and symptoms.

You must not be given Xenpozyme

• If you have experienced life-threatening allergic (anaphylactic) reactions to olipudase alfa (see section Warnings and precautions below) or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions
You may have side effects called infusion-associated reactions (IARs) that may be caused by the infusion (drip) of the medicine. They may occur while you are being given Xenpozyme or within 24 hours after the infusion.

They may include allergic reactions (see section 4) and symptoms such as headache, a raised, itchy rash (hives), fever, nausea, vomiting and itchy skin.

If you think you are having an IAR, tell your doctor straight away. If you have a severe allergic reaction during your infusion your doctor will stop your infusion and provide appropriate medical treatment. Your doctor will make a judgement about the risks and benefits of giving you further doses Xenpozyme.

If you have a mild or moderate IAR, your doctor or nurse may temporarily stop the infusion, lower the infusion rate, and/or reduce the dose.

Your doctor may also give (or have given) you other medicines to prevent or manage allergic reactions. Your doctor will order blood tests to check how well your liver is working (by measuring levels of your liver enzymes) before starting the treatment, and then at regular intervals as the doses are adjusted (see section 3).

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

Pregnancy and breast-feeding
If you are pregnant or breast-feeding, think you may be pregnant, or are planning to have a baby, ask your doctor or nurse for advice before using this medicine.

There is no experience with the use of Xenpozyme in pregnant women. Xenpozyme may be harmful to unborn children when taken by a woman during pregnancy. Xenpozyme should only be used during pregnancy if clearly necessary. Women who are able to become pregnant should use effective contraception during treatment and for 14 days after the last dose if Xenpozyme is discontinued.

It is not known whether Xenpozyme passes into human breast milk. Xenpozyme was detected in animal milk. Tell your doctor if you are breast-feeding or plan to do so. Your doctor will then help you decide whether to stop breast-feeding, or whether to stop taking Xenpozyme, considering the benefit of breast-feeding the baby and the benefit of Xenpozyme to the mother.

Driving and using machines
Xenpozyme may have a minor influence on the ability to drive and use machines because you may experience low blood pressure (which may make you feel faint).

Xenpozyme contains sodium
This medicine contains 0.60 mg sodium (main component of cooking/table salt) per 4 mg vial or 3.02 mg sodium per 20 mg vial. This is equivalent to 0.03% and 0.15%, respectively, of the recommended maximum daily dietary intake of sodium for an adult or an adolescent and 0.08% and 0.38%, respectively, of the maximum acceptable daily intake of sodium for children below 16 years of age.

Xenpozyme will be given to you as a drip (infusion) under the supervision of a healthcare professional who is experienced in the treatment of ASMD or other metabolic diseases.

The dose you receive is based on your body weight and will be given to you every two weeks. Treatment starts with a low dose of the medicine, which is gradually increased. Infusion usually lasts around 3 to 4 hours but may be shorter or longer based on your doctor s judgement, and may be shorter during the period whilst your dose is being increased.

Adult patients The recommended starting dose of Xenpozyme is 0.1 mg for each kg of body weight. This is increased in a planned way with each subsequent dose, until the recommended dose of 3 mg for each kg of body weight every 2 weeks is reached. It typically takes up to 14 weeks to reach the recommended dose but may be longer based on your doctor s judgement.

Children The recommended starting dose of Xenpozyme is 0.03 mg for each kg of body weight. The subsequent doses should be increased in a planned way up to the recommended dose of 3 mg for each kg of body weight every 2 weeks. It typically takes up to 16 weeks to reach the recommended dose but may be longer based on your doctor s judgement.

Home infusion Your doctor may consider home infusion of Xenpozyme if you are on stable dose and tolerating your infusions well. This decision to move to home infusion should be made after evaluation and recommendation by your doctor. If you get a side effect during an infusion of Xenpozyme, the person giving your home infusion may stop the infusion and start appropriate medical treatment.

Instructions for proper use Xenpozyme is given by intravenous infusion (a drip into a vein). It is supplied as a powder that will be mixed with sterile water before it is given.

If you are given more Xenpozyme than you should
Tell your doctor immediately, if you suspect a change from your routine infusion.

If you miss a Xenpozyme infusion
It is important to have your infusion every 2 weeks. An infusion is considered missed if not given within 3 days from the scheduled infusion. Depending on the number of missed doses, your doctor may have to restart from a lower dose.

If you have missed an infusion or are unable to attend a scheduled appointment, please contact your doctor right away.

If you have any further questions on the use of this medicine, ask your doctor or nurse.

Like all medicines, this medicine can cause side effects, although not everybody gets them. Infusion-associated reactions (IARs) have been seen while patients were being given the medicine or within 24 hours after the infusion.

The most serious side effects may include sudden severe allergic reactions, raised, itchy rash (hives), rash, increased liver enzymes and irregular heartbeat.

You must tell your doctor immediately if you get an IAR or an allergic reaction. If you have an infusion reaction you may be given additional medicines to treat or help prevent future reactions. If the infusion reaction is severe, your doctor may stop the infusion of Xenpozyme and start giving appropriate medical treatment.

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

• Headache
• Fever body temperature increased
• Raised, itchy rash (hives)
• Nausea
• Vomiting
• Abdominal (belly) pain
• Muscles aches
• Itchy skin
• Increased blood test for inflammation

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

• Rash (different types of rash sometimes with itch)
• Pain in upper belly
• Fatigue
• Abnormal blood test for liver function
• Diarrhoea
• Reddening of the skin
• Joint pain
• Back pain
• Chills
• Difficulty breathing
• Abdominal discomfort
• Bone pain
• Pain
• Low blood pressure
• Forceful heartbeat that may be rapid or irregular
• Fast heartbeat
• Liver pain
• Severe allergic reactions
• Feeling very warm
• Throat and voice box irritation
• Throat tightness and swelling
• Wheezing
• Skin lesions (such as solid elevated or red flat lesions)
• Rapid swelling under the skin in areas such as the face, throat, arms and legs which can be life threatening if throat swelling blocks the airway
• Stomach pain
• Itchy or red eyes
• Eye discomfort
• Weakness
• Abnormal blood test for inflammation
• Catheter site-related reactions including pain, itching, or swelling

Reporting of side effects
If you get any side effects, talk to your doctor or nurse. 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.

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

Do not use Xenpozyme after the expiry date stated on the label and the carton. The expiry date refers to the last day of the month.

Store in refrigerator between 2 C to 8 C.

After dilution, immediate use is recommended.

If not used immediately, the reconstituted solution may be stored for up to 24 hours at 2 C to 8 .

After dilution, the solution can be stored for up to 24 hours at 2-8 C followed by 12 hours (including infusion time) at room temperature.

Do not throw away any medicines via wastewater or household waste. Ask your doctor or nurse how to throw away medicines you no longer use. These measures will help protect the environment.

What Xenpozyme contains

• The active substance is olipudase alfa. One vial contains 4 mg or 20 mg of olipudase alfa.
• Other ingredients are
• L-methionine
• Sodium phosphate dibasic heptahydrate
• Sodium phosphate monobasic monohydrate
• Sucrose see section 2 Xenpozyme contains sodium

What Xenpozyme looks like and contents of the pack Xenpozyme is a powder for concentrate for solution for infusion in a vial (4 or 20 mg/vial). The powder is white to off-white lyophilised powder. After mixing with sterile water, it is a clear, colorless solution. The solution must be further diluted before infusion.

Marketing Authorisation Holder Sanofi B.V., Paasheuvelweg 25, 1105 BP Amsterdam, The Netherlands

Manufacturer Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

Belgi /Belgique/Belgien Sanofi Belgium T l/Tel: +32 (0)2 710 54 Lietuva Swixx Biopharma UAB Tel: +370 5 236 91
Swixx Biopharma EOOD .: +359 (0)2 4942 Luxembourg/Luxemburg Sanofi Belgium
T l/Tel: +32 (0)2 710 54 00 (Belgique/Belgien)

esk republika sanofi-aventis, s.r.o. Tel: +420 233 086 Magyarorsz g SANOFI-AVENTIS Zrt. Tel.: +36 1 505 0Danmark Sanofi A/S Tlf: +45 45 16 70 Malta Sanofi S.r.l. Tel: +39 02 39394Deutschland Sanofi-Aventis Deutschland GmbH Tel.: 0800 04 36 Tel. aus dem Ausland: +49 69 305 70 Nederland Sanofi B.V. Tel: + 31 20 245 4Eesti Norge Swixx Biopharma O
Tel: +372 640 10 sanofi-aventis Norge AS Tlf: +47 67 10 71
Sanofi-Aventis AEBE : +30 210 900 16 sterreich sanofi-aventis GmbH Tel: +43 1 80 185 0

Espa a sanofi-aventis, S.A.
Tel: +34 93 485 94 Polska sanofi-aventis Sp. z o.o. Tel.: +48 22 280 00 France Sanofi Winthrop Industrie T l: 0 800 222 Appel depuis l tranger : +33 1 57 63 23 Portugal Sanofi - Produtos Farmac uticos, Lda Tel: +351 21 35 89 Hrvatska
Swixx Biopharma d.o.o. Tel: +385 1 2078 Rom nia Sanofi Romania SRL Tel: +40 (0) 21 317 31 Ireland sanofi-aventis Ireland Ltd. T/A SANOFI Tel: +353 (0) 1 403 56 Slovenija Swixx Biopharma d.o.o.
Tel: +386 1 235 51 sland Vistor hf. S mi: +354 535 7Slovensk republika Swixx Biopharma s.r.o. Tel: +421 2 208 33 Italia Sanofi S.r.l. Tel: 800 536Suomi/Finland Sanofi Oy Puh/Tel: +358 (0) 201 200
C.A. Papaellinas Ltd. : +357 22 741Sverige Sanofi AB Tel: +46 (0)8 634 50 Latvija Swixx Biopharma SIA
Tel: +371 6 616 47 United Kingdom (Northern Ireland) sanofi-aventis Ireland Ltd. T/A SANOFI Tel: +44 (0) 800 035 2This leaflet was last revised in

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency web site: