ADZYNMA 500 IU Порошок и раствор для инъекционного раствора

Содержание инструкции

1. ADZYNMA 500 IU powder and solvent for injectable solution, 1 500 IU powder and solvent for injectable solution
   1. What is ADZYNMA and what is it used for
   2. What you need to know before you use ADZYNMA
   3. How to use ADZYNMA
   4. Possible side effects
   5. How to store ADZYNMA
   6. Package contents and other information
   7. Instructions for Use

ADZYNMA 500 IU powder and solvent for injectable solution, 1 500 IU powder and solvent for injectable solution

rADAMTS13

Medicinal product subject to additional monitoring. This will allow the rapid identification of new safety information. You can contribute by reporting any adverse effects encountered during the use of this medicine. See the end of section 4 for information on how to report adverse effects.

Read this leaflet carefully before you start using 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, pharmacist or nurse.
• This medicine has been prescribed for you only. Do not pass it on to others, even if their symptoms are the same as yours, as it could be harmful.
• If you get any side effects, even those not listed in this leaflet, tell your doctor, pharmacist or nurse. See section 4.

Contents of this leaflet

• 1. What ADZYNMA is and what it is used for
• 2. What you need to know before you use ADZYNMA
• 3. How to use ADZYNMA
• 4. Possible side effects
• 5. How to store ADZYNMA
• 6. Contents of the pack and other information
• 7. Instructions for use

1. What is ADZYNMA and what is it used for

ADZYNMA contains the active substance rADAMTS13, which is an artificial copy of the natural enzyme
(protein) ADAMTS13. This enzyme is absent in people with congenital thrombotic thrombocytopenic purpura (cTTP).
Congenital TTP is a very rare hereditary blood disease due to which blood clots form
in the small blood vessels throughout the body. These clots can block the flow
of blood and oxygen to the organs and this determines the presence in the blood of a number of platelets
(components that help the blood to coagulate) lower than normal.
Congenital TTP is caused by the absence of the ADAMTS13 enzyme in the blood. ADAMTS13 helps to
prevent the formation of blood clots by breaking down large molecules called von Willebrand factor (VWF). When VWF molecules are too large, they can cause dangerous blood clots. ADZYNMA is used to reconstitute the levels of the missing ADAMTS13 enzyme
and this allows the breakdown of these larger molecules into smaller molecules, reducing the
likelihood of blood clot formation and potentially preventing low platelet levels
in the blood in patients with cTTP.

2. What you need to know before you use ADZYNMA

Do not use ADZYNMA

• if you have had severe or potentially life-threatening allergic reactions to rADAMTS13 or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions
Talk to your doctor, pharmacist or nurse before using ADZYNMA.

Allergic reactions
There is a risk that you may experience an allergic-type hypersensitivity reaction to ADZYNMA.
Your doctor should inform you about the first signs of severe allergic reactions such as:

• increased heart rate
• chest tightness
• wheezing and/or sudden difficulty breathing
• low blood pressure
• hives, rash and itchy skin
• runny or congested nose
• red eyes
• sneezing
• rapid swelling under the skin in areas such as the face, throat, arms and legs
• fatigue
• nausea (feeling sick)
• vomiting
• feeling of numbness, tingling and pins and needles
• restlessness
• anaphylaxis (a severe allergic reaction that can cause difficulty swallowing and/or breathing and a red or swollen face and/or hands).

If any of these symptoms occur, your doctor will decide whether treatment with ADZYNMA
should be stopped and will give you appropriate medicines to treat the allergic reaction. Severe
symptoms, including difficulty breathing and dizziness, require prompt emergency treatment.

Inhibitors
Some patients treated with ADZYNMA may develop neutralising antibodies (called
inhibitors). These inhibitors may potentially prevent the treatment from working correctly.
Tell your doctor if you think the medicine is not working for you.

Other medicines and ADZYNMA
Tell your doctor or pharmacist if you are taking, have recently taken or might take
any other medicines.

Pregnancy and breastfeeding
If you are pregnant, suspect you are pregnant or are planning to become pregnant, or if you are
breastfeeding, ask your doctor or pharmacist for advice before taking this medicine.
You should not receive ADZYNMA during pregnancy unless your doctor specifically recommends it.
If you are breastfeeding, you and your doctor will need to decide whether you can use ADZYNMA.

Driving and using machines
This medicine may slightly impair the ability to drive and operate machines. In
following the use of ADZYNMA, dizziness and drowsiness may occur.

Traceability
In order to improve the traceability of biological medicines, the name and batch number of the
medicine must be clearly recorded.

ADZYNMA contains sodium
This medicine contains less than 1 mmol (23 mg) of sodium per vial, i.e. essentially
“sodium-free”.

ADZYNMA contains polysorbate 80
This medicine contains 2.7 mg of polysorbate 80 per vial of ADZYNMA 500 or
ADZYNMA 1 500 equivalent to up to 0.216 mg/kg. Polysorbates can cause allergic
reactions. Tell your doctor if you have known allergies.

3. How to use ADZYNMA

Treatment with ADZYNMA will be administered to you under the supervision of a doctor experienced in the
care of patients with blood diseases.
ADZYNMA is administered via intravenous injection (into a vein). It is provided to your doctor
in the form of a powder that will be dissolved (reconstituted) before administration with the provided
solvent (a liquid capable of dissolving the powder).
The dose is calculated based on body weight.
Taking the medicine at home
If you tolerate the injections well, your doctor may decide that you can use ADZYNMA at home.
When you are able to inject ADZYNMA yourself (or if it is administered to you by a caregiver) after
adequate training by your doctor and/or nurse, the doctor will continue to
monitor your response to treatment. If you experience undesirable effects when taking the medicine
at home, you should immediately stop the injection and contact a healthcare professional.
Recommended dose

Preventive enzymatic replacement therapy
The usual dose is 40 IU per kg of body weight, administered every other week.
If ADZYNMA every other week is not effective for you, your doctor may modify the frequency
to once a week.

On-demand enzymatic replacement therapy for sudden episodes of TTP
If you develop a sudden episode of thrombotic thrombocytopenic purpura (TTP), the recommended dose
of ADZYNMA is as follows:

• 40 IU/kg of body weight on day 1.
• 20 IU/kg of body weight on day 2.
• 15 IU/kg of body weight starting on day 3 once a day for two days after the resolution of the sudden episode of TTP.

If you take more ADZYNMA than you should
An overdose of this medicine can cause bleeding.

If you forget to use ADZYNMA
If you have missed an ADZYNMA injection, inform your doctor as soon as possible. Do not use a
double dose to make up for a missed dose.

If you stop taking ADZYNMA
If you wish to stop treatment with ADZYNMA, contact your doctor. If you stop the
treatment, the symptoms of the disease may worsen.
If you have any questions about the use of this medicine, ask your doctor, pharmacist or
nurse.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.
With ADZYNMA the following side effects have been reported:

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

• nasal and throat infection
• headache
• dizziness
• migraine
• diarrhoea
• nausea

Common(may affect up to 1 in 10 people)

• high number of platelets in the blood (thrombocytosis)
• somnolence
• constipation
• bloated stomach (abdominal distension)
• weakness (asthenia)
• feeling of warmth
• abnormal ADAMTS13 activity

Reporting of side effects
If you get any side effects, even those not listed in this leaflet, tell your doctor, pharmacist or nurse. You can also report side effects directly
through the national reporting system listed in Annex V. By reporting side effects you can help to provide more information on the safety of this medicine.

5. How to store ADZYNMA

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date which is stated on the label and the packaging after
Exp. The expiry date refers to the last day of that month.
Unopened vials
Store in a refrigerator (2 °C − 8 °C).
Do not freeze.
Store in the original packaging to protect the medicine from light.
Unopened ADZYNMA powder vials may be stored at room temperature
(up to 30 °C) for a maximum period of 6 months, without exceeding the expiry date. Do not return
ADZYNMA to the refrigerator after storing it at room temperature. Record on the packaging the
date on which ADZYNMA was removed from the refrigerator.
After reconstitution
Discard any unused reconstituted product after 3 hours.
Do not use this medicine if you notice that it is not clear and colourless.
Do not dispose of any medicine in 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 ADZYNMA contains

• The active substance, rADAMTS13, is a recombinant human “A-disintegrin and metalloproteinase with thrombospondin motif 13” purified. Each vial of powder contains 500 or 1,500 IU of nominal rADAMTS13 activity.
• The solvent vial contains 5 mL of water for injection.
• The other excipients are sodium chloride, calcium chloride dihydrate, L-histidine, mannitol, sucrose and polysorbate 80 (E433). See section 2 “ADZYNMA contains sodium” and “ADZYNMA contains polysorbate 80”.

Description of the appearance of ADZYNMA and contents of the pack
ADZYNMA is supplied as powder and solvent for solution for injection. The powder is a
white lyophilized powder. The solvent is clear and colorless.
Each pack contains one vial of powder, one vial of solvent, a reconstitution device (BAXJECT II Hi-Flow), one single-use syringe, an infusion set and two alcohol-soaked swabs.

Marketing authorisation holder and manufacturer
Takeda Manufacturing Austria AG
Industriestrasse 67
1221 Vienna
Austria
For further information on this medicinal product, contact the local representative of the marketing
authorisation holder:

België/Belgique/Belgien
Takeda Belgium NV
Tél/Tel: +32 2 464 06 11
[email protected]

Lietuva
Takeda, UAB
Tel: +370 521 09 070
[email protected]

България
Такеда България ЕООД
Тел.: +359 2 958 27 36
[email protected]

Luxembourg/Luxemburg
Takeda Belgium NV
Tél/Tel: +32 2 464 06 11
[email protected]

Česká republika
Takeda Pharmaceuticals Czech Republic s.r.o.
Tel: +420 234 722 722
[email protected]

Magyarország
Takeda Pharma Kft.
Tel.: +36 1 270 7030
[email protected]

Danmark
Takeda Pharma A/S
Tlf.: +45 46 77 10 10
[email protected]

Malta
Τakeda HELLAS S.A.
Tel: +30 210 6387800
[email protected]

Deutschland
Takeda GmbH
Tel: +49 (0)800 825 3325
[email protected]

Nederland
Takeda Nederland B.V.
Tel: +31 20 203 5492
[email protected]

Eesti
Takeda Pharma OÜ
Tel: +372 6177 669
[email protected]

Norge
Takeda AS
Tlf: +47 800 800 30
[email protected]

Ελλάδα
Τakeda ΕΛΛΑΣ Α.Ε.
Τηλ: +30 210 6387800
[email protected]

Österreich
Takeda Pharma Ges.m.b.H.
Tel: +43 (0) 800-20 80 50
[email protected]

España
Takeda Farmacéutica España, S.A.
Tel: +34 917 90 42 22
[email protected]

Polska
Takeda Pharma Sp. z o.o.
Tel.: +48223062447
[email protected]

France
Takeda France SAS
Tél: + 33 1 40 67 33 00
[email protected]

Portugal
Takeda Farmacêuticos Portugal, Lda.
Tel: + 351 21 120 1457
[email protected]

Hrvatska
Takeda Pharmaceuticals Croatia d.o.o.
Tel: +385 1 377 88 96
[email protected]

România
Takeda Pharmaceuticals SRL
Tel: +40 21 335 03 91
[email protected]

Ireland
Takeda Products Ireland Ltd
Tel: 1800 937 970
[email protected]

Slovenija
Takeda Pharmaceuticals farmacevtska družba d.o.o.
Tel: + 386 (0) 59 082 480
[email protected]

Ísland
Vistor hf.
Sími: +354 535 7000
[email protected]

Slovenská republika
Takeda Pharmaceuticals Slovakia s.r.o.
Tel: +421 (2) 20 602 600
[email protected]

Italia
Takeda Italia S.p.A.
Tel: +39 06 502601
[email protected]

Suomi/Finland
Takeda Oy
Puh/Tel: 0800 774 051
[email protected]

Κύπρος
Τakeda ΕΛΛΑΣ Α.Ε.
Τηλ: +30 210 6387800
[email protected]

Sverige
Takeda Pharma AB
Tel: 020 795 079
[email protected]

Latvija
Takeda Latvia SIA
Tel: +371 67840082
[email protected]
An authorisation has been granted for this medicinal product in “exceptional circumstances”. This means
that, due to the rarity of the disease, complete information on this medicinal product has not been possible
to obtain. The European Medicines Agency will annually review any new information on this

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. Additionally, links to other websites on rare diseases and
related therapeutic treatments are provided.

7. Instructions for Use

These instructions for use contain information on how to reconstitute and infuse ADZYNMA.

These instructions for use are intended for healthcare professionals and patients/caregivers who
will administer ADZYNMA at home after adequate training by a healthcare
professional.
Treatment with ADZYNMA must be prescribed and supervised by a healthcare professional
experienced in the care of patients with blood diseases.

Important:

• For intravenous injection only after reconstitution.
• Use aseptic technique throughout the procedure.
• Check the product expiry date before use.
• Do notuse ADZYNMA if the expiry date has been exceeded.
• If the patient requires more than one vial of ADZYNMA per injection, reconstitute each vial according to the instructions in the “Reconstitution” section.
• Before administration, inspect the reconstituted ADZYNMA solution for the presence of particles and colour change. The solution should appear clear and colourless.
• Do notadminister if particles or colour change are observed.
• Use ADZYNMA within3 hoursof reconstitution if stored at room temperature.
• Do notadminister ADZYNMA in the same line or container simultaneously with other infusion medicines.

Reconstitution

• 1. Prepare a flat, clean surface and gather all necessary materials for reconstitution and administration ( Figure A).

Figure A

Materials for reconstitutionMaterials for administration

• 2. Allow the ADZYNMA vials and the diluent to reach room temperature before use.
• 3. Wash and dry your hands thoroughly.

• 4. Remove the plastic caps from the ADZYNMA and diluent vials and place the vials on a flat surface ( Figure B).

Figure B

• 5. Rub the rubber stoppers with a cotton swab soaked in alcohol and allow them to dry before use. ( Figure C).

Figure C

• 6. Open the BAXJECT II Hi-Flow device packaging by removing the cover, without touching the inside ( Figure D).
• Do notremove the BAXJECT II Hi-Flow device from the packaging.
• Do nottouch the transparent plastic perforator.

Figure D

• 7. Invert the packaging with the BAXJECT II Hi-Flow device and position it over the diluent vial. Press down until the transparent plastic perforatorpunctures the stopper of the diluent vial( Figure E).

Figure E

• 8. Grab the BAXJECT II Hi-Flow device packaging by the edge and remove the packaging from the device ( Figure F).
• Do notremove the blue capfrom the BAXJECT II Hi-Flow device.
• Do nottouch the exposed purple plastic perforator.

Figure F

• 9. Invert the systemso that the diluent vialis now on top. Press the BAXJECT II Hi-Flow device down until the purple plastic perforatorpunctures the stopper of the ADZYNMA powder vial( Figure G). The vacuum will draw the diluent into the ADZYNMA powder vial.
• Bubbles or foam may be noticed: this is normal and should disappear shortly.

Figure G

Purple plastic
Powder

• 10. Gently and continuously swirl the connected vials until the powder is completely dissolved ( Figure H).
• Do notshake the vial.

Figure H

• 11. Visually inspect the reconstituted solution for particles before administration.
• Do notuse the product if particles or colour change are observed.
• 12. If the dose requires more than one vial of ADZYNMA, reconstitute each vial following the above steps.
• Use a different BAXJECT II Hi-Flow device to reconstitute eachADZYNMA and diluent vial.

Administration of ADZYNMA

• 13. Remove the blue capfrom the BAXJECT II Hi-Flow device ( Figure I). Attach a Luer-Lock syringe ( Figure J).
• Do notinject air into the system. Figure IFigure J

• 14. Invert the system(now the ADZYNMA vial is on top). Draw the reconstituted solutioninto the syringe by slowly pulling back the plunger ( Figure K).

Figure K

• 15. If a patient needs to receive more than one vial of ADZYNMA, the contents of multiple vials can be drawn into the same syringe. Repeat this procedure for all reconstituted ADZYNMA vials until the total volume to be administered is reached.
• 16. Disconnect the syringe and attach a suitable injection needle or infusion set.
• 17. Point the needle upwards and remove any air bubbles by gently tapping the syringe with your finger and slowly and carefully pushing the air out of the syringe and needle.
• 18. Apply a tourniquet and clean the chosen injection site with an alcohol swab ( Figure L).

Figure L

• 19. Insert the needle into the vein and remove the tourniquet.
• 20. Infuse ADZYNMA reconstituted slowlyat a rate of 2- 4 mL per minute( Figure M).
• A syringe pump can be used to control the infusion rate.

Figure M

• 21. Remove the needle from the vein and apply pressure to the injection site for several minutes.
• Do notreplace the cap on the needle.

Storage of ADZYNMA

• Store ADZYNMA in a refrigerator (2 °C - 8 °C) or at room temperature (up to 30 °C) for a maximum period of 6 months.
• Do notstore ADZYNMA in the refrigerator after storing it at room temperature.
• Recordon the box the date when ADZYNMA was removed from the refrigerator.
• Do notfreeze.
• Store in the original packaging to protect the medicine from light.
• Do notuse beyond the expiry date stated on the label and the carton after Exp.
• Use ADZYNMA within3 hoursof reconstitution. Discard any reconstituted product if not used within 3 hours of reconstitution.

Disposal of ADZYNMA

• The vials are for single useonly.
• Dispose of the used needle, syringe and vials in a puncture-resistant sharps container.
• Do not dispose of any medicine in the wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

APPENDIX IV
CONCLUSIONS RELATING TO THE GRANT OF THE MARKETING AUTHORISATION
IN EXCEPTIONAL CIRCUMSTANCES PRESENTED BY THE EUROPEAN MEDICINES
AGENCY
Conclusions presented by the European Medicines Agency on:

• Grant of marketing authorisation in exceptional circumstancesFollowing the evaluation of the marketing authorisation application, the Committee for Medicinal Products for Human Use ( Committee for human medicinal products,CHMP) considers that the benefit/risk balance is favourable in order to recommend the grant of a marketing authorisation in exceptional circumstances, as further described in the European Public Assessment Report ( European Public Assessment Report, EPAR).