Xenpozyme 20 mg powder for concentrate for solution for infusion
SpainTable of Contents
- Package leaflet: Information for the user
- Introduction
- 1. What Xenpozyme is and what it is used for
- 2. What you need to know before Xenpozyme is administered to you
- 3. How Xenpozyme is administered
- 4. Possible adverse effects
- 5. Storage of Xenpozyme
- 6. Contents of the container and other information
Package leaflet: Information for the user
Introduction
Package leaflet: information for the patient
Xenpozyme 4 mg powder for concentrate for solution for infusion
Xenpozyme 20 mg powder for concentrate for solution for infusion
olipudase alfa
This medicinal product is subject to additional monitoring, which will allow for rapid identification of new safety information. You can help by reporting any side effects you may experience. Information on how to report side effects is included at the end of section 4.
Read the entire leaflet carefully before starting to use this medicine, as it contains important information for you.
- Keep this leaflet, as you may need to read it again.
- If you have any questions, consult your doctor or nurse.
- If you experience any side effects, consult your doctor or nurse, even if they are side effects not listed in this leaflet. See section 4.
Leaflet contents
- What Xenpozyme is and what it is used for
- What you need to know before being administered Xenpozyme
- How Xenpozyme is administered
- Possible side effects
- How to store Xenpozyme
- Contents of the pack and other information
1. What Xenpozyme is and what it is used for
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 signs and symptoms of non-central nervous system (non-CNS) ASMD.
How Xenpozyme works
Patients with ASMD lack a properly functioning version of the enzyme acid sphingomyelinase. This leads to the accumulation of a substance called sphingomyelin, which damages organs such as the spleen, liver, heart, lungs, and blood. Olipudase alfa works in the same way as the natural enzyme would, acting as a replacement and thereby reducing the accumulation of sphingomyelin in organs and treating the associated signs and symptoms.
2. What you need to know before Xenpozyme is administered to you
You must not be given Xenpozyme:
- If you have experienced potentially life-threatening allergic (anaphylactic) reactions to olipudase alfa (see section “Warnings and precautions” below) or to any of the other components of this medicine (listed in section 6).
Warnings and precautions
You may experience adverse effects called infusion-associated reactions (IARs), which may be caused by the infusion (drip) of the medicine. These may occur during or within 24 hours after the infusion of Xenpozyme.
They may include allergic reactions (see section 4) and symptoms such as headache, raised itchy skin rash (wheals), fever, nausea, vomiting, and skin itching.
If you think you are experiencing an IAR, inform your doctor immediately.
If you have a severe allergic reaction during the infusion, your doctor will stop the infusion and provide appropriate medical treatment. Your doctor will then decide on the risks and benefits of administering further doses of Xenpozyme.
If you have a mild or moderate IAR, your doctor or nurse may temporarily stop the infusion, reduce the infusion rate, and/or reduce the dose.
Your doctor may also administer (or may have already administered) other medications to prevent or manage allergic reactions.
Your doctor will order blood tests to monitor your liver function (by measuring your liver enzyme levels) before starting treatment and then at regular intervals as doses are adjusted (see section 3).
Other medicines and Xenpozyme
Tell your doctor or nurse if you are using, have recently used, or might need to 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 or nurse before using this medicine.
Experience with the use of Xenpozyme in pregnant women is limited. Xenpozyme may be harmful to the fetus if taken by a woman during pregnancy. Xenpozyme should only be used during pregnancy if clearly necessary. Women of childbearing potential must use effective contraception during treatment and for 14 days after the last dose if Xenpozyme is discontinued.
It is unknown whether Xenpozyme passes into human breast milk. Xenpozyme has been detected in animal milk. Inform your doctor if you are breastfeeding or plan to breastfeed. Your doctor will then help you decide whether to discontinue breastfeeding or the administration of Xenpozyme, after considering the benefit of breastfeeding for the child and the benefit of Xenpozyme for the mother.
Driving and using machines
Xenpozyme may have a minor influence on the ability to drive and use machines, as you may experience a drop in blood pressure (which may make you feel dizzy).
Xenpozyme contains sodium
This medicine contains 0.60 mg of sodium (a main component of table/cooking salt) per 4 mg vial or 3.02 mg of sodium per 20 mg vial. This corresponds to 0.03% and 0.15%, respectively, of the maximum recommended daily intake of sodium for an adult or adolescent, and < 0.08% and < 0.38%, respectively, of the maximum acceptable daily sodium intake for children under 16 years of age.
3. How Xenpozyme is administered
Xenpozyme will be given to you as an intravenous infusion (drip) under the supervision of a healthcare professional experienced in the treatment of ASMD or other metabolic disorders.
The dose you receive is based on your body weight and will be administered every two weeks.
Treatment is started with a low dose of the medicine, which is gradually increased.
The infusion usually takes about 3 to 4 hours; however, it may be shorter or longer at the discretion of your doctor, and may be shorter during the dose escalation period.
Adult patients
The recommended starting dose of Xenpozyme is 0.1 mg per kg of body weight. This dose will be systematically increased with each subsequent infusion until reaching the recommended dose of 3 mg per kg of body weight every two weeks. It usually takes up to 14 weeks to reach the recommended dose, but this period may be longer at the discretion of your doctor.
Children
The recommended starting dose of Xenpozyme is 0.03 mg per kg of body weight. Subsequent doses should be systematically increased until reaching the recommended dose of 3 mg per kg of body weight every two weeks. It usually takes up to 16 weeks to reach the recommended dose, but this period may be longer at the discretion of your doctor.
Home infusion
Your doctor may consider home infusion of Xenpozyme if you are receiving a stable dose and are tolerating your infusions well. The decision to switch to home infusion should be made following evaluation and recommendation by your doctor. If you experience an adverse reaction during a Xenpozyme infusion, the person administering the infusion at home may stop the infusion and initiate appropriate medical treatment.
Instructions for proper use
Xenpozyme is administered by intravenous infusion (a drip into a vein). It is supplied as a powder that will be mixed with sterile water before administration.
If you receive more Xenpozyme than you should
Inform your doctor immediately if you notice any changes from your usual infusion.
If you miss an infusion of Xenpozyme
It is important that you receive the infusion every two weeks. An infusion is considered missed if it is not administered within 3 days after the scheduled infusion date. Depending on the number of missed doses, your doctor may need to restart treatment at a lower dose.
If you have missed an infusion or are unable to attend a scheduled appointment, contact your doctor immediately.
If you have any further questions about the use of this medicine, ask your doctor or nurse.
4. Possible adverse effects
Like all medicines, this medicine can cause adverse effects, although not everyone experiences them.
Infusion-related reactions (IRRs) have been observed while patients were receiving the medicine or within 24 hours after the infusion.
The most serious adverse effects may include sudden severe allergic reactions, raised itchy rash (hives), rash, increased liver enzymes, and irregular heartbeats.
You should inform your doctor immediately if you experience an IRR or an allergic reaction.
If you have a reaction to the infusion, you may be given additional medicines to treat or help prevent future reactions. If the infusion reaction is severe, your doctor may stop the Xenpoxyme infusion and start appropriate medical treatment.
Very common (may affect more than 1 in 10 people):
- Headache
- Raised itchy rash (hives)
- Fever – high body temperature
- Nausea
- Abdominal (stomach) pain
- Vomiting
- Itching of the skin
- Muscle pain
- Rash
- Blood tests showing increased values due to inflammation
- Upper abdominal pain
- Redness of the skin
Common (may affect up to 1 in 10 people):
- Red or itchy eyes
- Joint pain
- Fatigue
- Abnormal blood tests due to liver function
- Diarrhea
- Low blood pressure
- Difficulty breathing
- Abdominal discomfort
- Rash (different types of rash, sometimes itchy)
- Back pain
- Pain
- Chills
- Feeling very hot
- Wheezing (difficulty breathing)
- Irritation of the throat and larynx
- Liver pain
- Skin lesions (such as raised or flat red spots)
- Bone pain
- Weakness
- Severe allergic reactions
- Eye discomfort
- Rapid heartbeat
- Strong heartbeats that may be fast or irregular
- Tightness and swelling of the throat
- Stomach pain
- Rapid swelling under the skin in areas such as the face, throat, arms, and legs, which can be life-threatening if swelling in the throat blocks the airways
- Reactions related to the catheter site, including pain, itching, or swelling
- Abnormal blood tests due to inflammation
Reporting of adverse effects
If you experience any adverse effect, consult your doctor or nurse, even if it is a possible adverse effect not listed in this leaflet. You may also report them directly through the national reporting system included in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.
5. Storage of Xenpozyme
Keep this medicine out of the sight and reach of children.
Do not use Xenpozyme after the expiry date stated on the label and packaging. The expiry date refers to the last day of the month indicated.
Store in a refrigerator between 2 °C and 8 °C.
After dilution, it is recommended to use immediately.
If not used immediately, the reconstituted solution can be stored for up to 24 hours at a temperature between 2 °C and 8 °C.
After dilution, the solution may be stored for up to 24 hours at 2 °C to 8 °C, followed by up to 12 hours (including infusion time) at room temperature.
Medicines must not be disposed of via wastewater or household waste. Ask your doctor or nurse how to dispose of medicines and containers you no longer need. This will help protect the environment.
6. Contents of the container and other information
Composition of Xenpozyme
The active substance is olipudase alfa. One vial contains 4 mg or 20 mg of olipudase alfa.
The other components are:
- L-methionine
- Disodium phosphate heptahydrate
- Monosodium phosphate monohydrate
- Sucrose
see section 2 Xenpozyme contains sodium
Appearance of the product and contents of the container
Xenpozyme is a powder for concentrate for solution for infusion in a vial (4 or 20 mg/vial).
The powder is a white to off-white lyophilized powder.
After mixing with sterile water, it forms a clear, colourless solution. The solution must be further diluted before infusion.
Marketing Authorization Holder
Sanofi B.V., Paasheuvelweg 25, 1105 BP Amsterdam, The Netherlands
Manufacturer
Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland
For further information about this medicinal product, please contact the local representative of the Marketing Authorization Holder:
Belgium/Belgium/Belgium Sanofi Belgium Tel/Tel: +32 (0)2 710 54 00 | Lithuania Swixx Biopharma UAB Tel: +370 5 236 91 40 |
| Luxembourg/Luxembourg Sanofi Belgium Tél/Tel: +32 (0)2 710 54 00 (Belgium/Belgium) |
Czech Republic sanofi-aventis, s.r.o. Tel: +420 233 086 111 | Hungary SANOFI-AVENTIS Zrt. Tel.: +36 1 505 0050 |
Denmark Sanofi A/S Tlf: +45 45 16 70 00 | Malta Sanofi S.r.l. Tel: +39 02 39394275 |
Germany Sanofi-Aventis Deutschland GmbH Tel.: 0800 04 36 996 Tel. from abroad: +49 69 305 70 13 | Netherlands Sanofi B.V. Tel: +31 20 245 4000 |
Estonia Swixx Biopharma OÜ Tel: +372 640 10 30 | Norway sanofi-aventis Norge AS Tlf: +47 67 10 71 00 |
Greece Sanofi-Aventis Monoprosopi EPE Tel: +30 210 900 16 00 | Austria sanofi-aventis GmbH Tel: +43 1 80 185 – 0 |
Spain sanofi-aventis, S.A. Tel: +34 93 485 94 00 | Poland sanofi-aventis Sp. z o.o. Tel.: +48 22 280 00 00 |
France Sanofi Winthrop Industrie Tél: 0 800 222 555 Call from abroad: +33 1 57 63 23 23 | Portugal Sanofi - Produtos Farmacêuticos, Lda Tel: +351 21 35 89 400 |
Croatia Swixx Biopharma d.o.o. Tel: +385 1 2078 500 | Romania Sanofi Romania SRL Tel: +40 (0) 21 317 31 36 |
Ireland sanofi-aventis Ireland Ltd. T/A SANOFI Tel: +353 (0) 1 403 56 00 | Slovenia Swixx Biopharma d.o.o. Tel: +386 1 235 51 00 |
Iceland Vistor ehf. Tel: +354 535 7000 | Slovakia Swixx Biopharma s.r.o. Tel: +421 2 208 33 600 |
Italy Sanofi S.r.l. Tel: 800 536389 | Finland Sanofi Oy Puh/Tel: +358 (0) 201 200 300 |
Cyprus C.A. Papaellinas Ltd. Tel: +357 22 741741 | Sweden Sanofi AB Tel: +46 (0)8 634 50 00 |
Latvia Swixx Biopharma SIA Tel: +371 6 616 47 50 |
Date of the most recent review of this summary:
Other sources of information
Detailed information on this medicine is available on the website of the European Medicines Agency: http://www.ema.europa.eu. Links to other websites on rare diseases and orphan medicinal products are also available.
This summary can be found on the European Medicines Agency's website in all languages of the European Union/European Economic Area, and on the website: www.xenpozyme.info.sanofi, or by scanning the QR code included below (also included on the outer packaging) with a smartphone.
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This information is intended for healthcare professionals only:
Preparation of the solution for administration
The powder for concentrate for infusion solution must be reconstituted with sterile water for injections, then diluted with sodium chloride 9 mg/ml (0.9%) solution for injection, and subsequently administered by intravenous infusion.
The reconstitution and dilution steps must be carried out under aseptic conditions. Filtration devices must not be used at any time during the preparation of the infusion solution. Avoid foaming during reconstitution and dilution steps.
- Determine the number of vials to be reconstituted based on the patient's individual weight and prescribed dose.
Patient weight (kg) × dose (mg/kg) = patient dose (in mg). For example, when using 20 mg vials, patient dose (in mg) divided by 20 mg/vial = number of vials to reconstitute. If the number of vials includes a fraction, round up to the next whole number.
- Remove the required number of vials from the refrigerator and allow them to reach room temperature for approximately 20 to 30 minutes.
- Reconstitute each vial by injecting:
1.1 ml of sterile water for injections into the 4 mg vial
5.1 ml of sterile water for injections into the 20 mg vial
using a slow, gentle stream directed against the inner wall of the vial.
- Gently tilt and rotate each vial. Each vial should yield a clear, colourless solution at a concentration of 4 mg/ml.
- Visually inspect the reconstituted solution in the vials for particles or discoloration. The Xenpozyme solution must be clear and colourless. Do not use any vial showing opaque particles or discoloration.
- Withdraw the volume of reconstituted solution corresponding to the prescribed dose from the appropriate number of vials and dilute with sodium chloride 9 mg/ml (0.9%) solution for injection in a syringe or infusion bag, depending on the required infusion volume (see Table 1 for recommended total infusion volumes based on patient age and/or weight).
Table 1: Recommended infusion volumes
Body weight ≥ 3 kg to < 10 kg | Body weight ≥ 10 kg to < 20 kg | Body weight ≥ 20 kg (pediatric patients < 18 years) | Adult patients (≥ 18 years) | |
Dose (mg/kg) | Total infusion volume (ml) | Total infusion volume (ml) | Total infusion volume (ml) | Total infusion volume (ml) |
0.03 | The variable volume will vary according to body weight | The variable volume will vary according to body weight | 5 | NP |
0.1 | The variable volume will vary according to body weight | 5 | 10 | 20 |
0.3 | 5 | 10 | 20 | 100 |
0.6 | 10 | 20 | 50 | 100 |
1.0 | 20 | 50 | 100 | 100 |
2.0 | 50 | 75 | 200 | 100 |
3.0 | 50 | 100 | 250 | 100 |
-
For variable final infusion volumes based on body weight in pediatric patients (see Table 1):
-
Prepare an infusion solution at 0.1 mg/mL by adding 0.25 mL (1 mg) of the reconstituted solution prepared in step 3) and 9.75 mL of sodium chloride 9 mg/mL (0.9%) injection solution into a 10 mL empty syringe.
-
Calculate the volume (mL) required to achieve the patient's dose (mg).
Example: 0.3 mg ÷ 0.1 mg/mL = 3 mL
-
Dilution instructions for total volume between 5 mL and 20 mL using a syringe:
-
Slowly inject the required volume of the reconstituted solution into the inner wall of an empty syringe.
-
Slowly add sufficient sodium chloride 9 mg/mL (0.9%) injection solution to obtain the required total infusion volume (avoid foaming within the syringe).
-
Dilution instructions for total volume ≥ 50 mL using an infusion bag:
-
Empty infusion bag:
- Slowly inject the required volume of the reconstituted solution from step 3) into a sterile infusion bag of appropriate size.
- Slowly add sufficient sodium chloride 9 mg/mL (0.9%) injection solution to obtain the required total infusion volume (avoid foaming within the bag).
-
Pre-filled infusion bag:
- Remove from the pre-filled infusion bag containing sodium chloride 9 mg/mL (0.9%) injection solution a volume of normal saline equal to that of the reconstituted solution to be added, so that the final volume corresponds to that specified in Table 1.
- Slowly add the required volume of the reconstituted solution from step 3) into the infusion bag (avoid foaming within the bag).
- Gently invert the syringe or infusion bag to mix. Do not shake. Because this is a protein solution, slight flocculation (described as thin translucent fibers) may occasionally occur after dilution.
- The diluted solution must be filtered through a low-protein-binding 0.2 μm in-line filter during administration.
After completion of the infusion, flush the infusion line with sodium chloride 9 mg/mL (0.9%) injection solution, using the same infusion rate as that used during the final part of the infusion.