Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - strimvelis

Document Subject

Generated Narrative: MedicinalProductDefinition mpffc23a76b4090b54b8bc958f8ff82cb2

identifier: http://ema.europa.eu/identifier/EU/1/16/1097/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Strimvelis 1-10 x 106 cells/mL dispersion for infusion

type: Full name

part

part: nan

type: Invented name part

part

part: nan

type: Scientific name part

part

part: nan

type: Strength part

part

part: nan

type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-ffc23a76b4090b54b8bc958f8ff82cb2

identifier: http://ema.europa.eu/identifier/EU/1/16/1097/001

status: Final

type: Package Leaflet

category: Raw

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

author: Organization ACME industry

title: TEST PURPOSES ONLY - strimvelis

Attesters

What is in this leaflet

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

Strimvelis is a type of medicine called a gene therapy. It is made specially for each patient.

Strimvelis is used in children to treat a serious condition called ADA-SCID (adenosine deaminase-severe combined immune deficiency). It is used when your child cannot receive a bone marrow transplant from a family donor because the match is not close enough.

ADA-SCID occurs because of a faulty gene in the blood cells of your child s immune system. As a result, the cells do not produce enough of an enzyme called adenosine deaminase (ADA) and your child s immune system does not work properly to defend the body against infections.

To make Strimvelis, stem cells from your child s bone marrow are modified in the laboratory to insert a gene that produces ADA. When these modified stem cells are given back to your child, they can divide to produce different types of blood cells, including cells involved in your child s immune system.

Strimvelis is not suitable for some people

Strimvelis must not be given if your child:

• is allergic to any of the ingredients of this medicine (listed in section 6)
• has or has had a type of cancer called leukaemia or myelodysplasia
• has tested positive for HIV or some other infections (your doctor will advise you about this)
• has already been treated with gene therapy

Warnings and precautions

Information about cell-based medicinal products, like Strimvelis, must be kept for 30 years at the hospital. The information kept about your child will be your child s name and the batch number of Strimvelis your child received.

Strimvelis is made specially from the patient s own cells. It must never be given to anyone else.

Inserting a new gene into the DNA could cause blood cancer. There has been a case of blood cancer, called leukaemia in one patient several years after treatment with Strimvelis. It is therefore important to monitor your child for the symptoms of leukaemia.
These include fever, shortness of breath, paleness, night sweats, tiredness, swollen lymph glands, frequent infections, a tendency to bleed and/or bruise easily, or tiny red or purple spots under the skin. If your child develops any of these symptoms you should contact your doctor immediately.

Before treatment with Strimvelis, your child will be given other medicines (see sections 3 and 4 for more information on these medicines, including possible side effects).

If your child has previously tested positive for hepatitis C, your child can still be treated under certain conditions. Your doctor will discuss this with you if needed.

Central venous catheters are thin, flexible tubes, that are inserted by a doctor into a large vein to access the bloodstream of your child. The risks of these lines are infections and the formation of blood clots. The doctor and nurses will monitor your child for any central venous catheter complications.

Treatment with Strimvelis has been unsuccessful in some patients. These patients received alternative treatment options.

There is a small risk of infection as a result of the treatment. Your child s doctors and nurses will monitor them throughout the infusion for signs of infection and provide treatment if needed.

Some patients can develop autoimmunity i.e. trigger an immune response against their own cells or tissues (see section 4). Your child s doctor will discuss this with you if needed.

After the treatment, your child must not donate blood, organs, tissues or cells at any time in future. This is because Strimvelis is a gene therapy product.

When Strimvelis treatment cannot be completed In some cases, it might not be possible to go ahead with the planned treatment with Strimvelis for reasons such as:

• a problem taking the cells from your child s bone marrow to make the medicine
• not enough of the right type of cells in the tissue taken from your child s body to make the medicine
• the medicine not meeting all the quality tests
• a delay in the medicine reaching the clinic where your child is being treated.

Before receiving Strimvelis your child will be given chemotherapy in order to remove their existing bone marrow. If Strimvelis cannot be administered after chemotherapy or if the modified stem cells do not take hold (engraft) in your child s body, the doctor will give your child replacement stem cells, using the backup sample that was collected and stored before treatment started (see also section 3, How Strimvelis is given).

You may need other treatment Strimvelis goes through a range of tests before it is used. Because it is given soon after it is made, the final results of some of these tests will not be ready before the medicine is given. If the tests show anything that might affect your child, the doctor will treat your child as appropriate.

Other medicines and Strimvelis
Tell your doctor if your child is taking, has recently taken or might take any other medicines.

Your child must not be given vaccines called live vaccines for 6 weeks before they are given the conditioning medicine to prepare for Strimvelis treatment, nor after treatment while your child s immune system is recovering.

Strimvelis contains sodium This medicine contains 42 to 137 mg sodium (main component of cooking/table salt) in each dose. This is equivalent to 2 to 7% of the recommended maximum daily dietary intake of sodium for an adult.

Strimvelis is given by a drip (infusion) into a vein (intravenously). It must be given in a specialised hospital, and by a doctor who is experienced in treating patients with ADA-SCID and in using this type of medicine.

Before Strimvelis is made, the doctor will do tests to make sure that your child is not carrying certain infections (see section 2).

Two samples are collected The doctor will collect two samples of bone marrow cells before the planned treatment:

• the backup sample, collected at least 3 weeks before Strimvelis treatment. It will be stored, to be given as replacement cells if Strimvelis cannot be given or does not work (see When Strimvelis treatment cannot be completed in section 2)
• the treatment sample, collected 4 to 5 days before Strimvelis treatment. It will be used to make Strimvelis, by inserting a new gene into the cells.

Before and during Strimvelis treatment

When What is done Why

At least 3 weeks before treatment

Backup sample of stem cells collected

to be stored as a backup (see above)

About 4 to 5 days before treatment

Treatment sample of stem cells collected

to make Strimvelis (see above)

3 days and 2 days before treatment

A medicine called busulfan is given 4 times a day for 2 days (total of 8 doses)

to prepare the bone marrow for Strimvelis treatment and clear existing stem cells

About 15 to 30 minutes before treatment

An antihistamine medicine may be given

to make it less likely that you will react to the infusion

Strimvelis is given
by a drip into a vein. This will take about 20 minutes

Like all medicines, this medicine can cause side effects, although not everybody gets them.
The side-effects linked to Strimvelis are caused by the immune system becoming over-active and attacking the body s own tissues. Some side effects may also be related to the busulfan medicine used to prepare your child s bone marrow for treatment; these are marked with an asterisk (*) in the list below.

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

• runny or blocked nose (allergic rhinitis)
• wheezing, difficulty breathing (asthma)
• inflamed itchy skin (atopic dermatitis, eczema)
• raised temperature (pyrexia)
• underactive thyroid gland (hypothyroidism)
• high blood pressure (hypertension)*
• decreases in the number of red or white blood cells (anaemia, neutropenia)*
• increases in liver enzymes (which indicate stress on the liver)* blood test results positive for antinuclear antibody and smooth muscle antibody (which might suggest possible autoimmunity)

Common: may affect up to 1 in 10 people.

• red or purple dots on the skin, bleeding under the skin (autoimmune thrombocytopenia)
• inflamed thyroid gland (autoimmune thyroiditis)
• weakness and pain in the feet and hands caused by damage to nerves (Guillain-Barr syndrome)
• inflamed liver (autoimmune hepatitis)
• reduced numbers of blood cells (autoimmune haemolytic anaemia, autoimmune aplastic anaemia)
• blood test results positive for antineutrophil cytoplasmic antibody (which could lead to autoimmune inflammation and swelling of the blood vessels and possibly increased level of infections)
• a type of blood cancer called leukaemia

If you have any questions about symptoms or side effects, or if any symptoms concern you, talk to your child s doctor or nurse.

Reporting of side effects If your child gets 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.

The following information is intended for doctors only.

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

Do not use this medicine after the expiry date and time (EXP) which is stated on the container label and infusion bag label.

Store at 15 30 C.

This medicine contains genetically-modified human cells. Unused medicine or waste material must be disposed of in compliance with the local guidelines on handling human-derived material. As this medicine will be given by a qualified doctor, they are responsible for correct disposal of the product. These measures will help protect the environment.

What Strimvelis contains

• The active substance is an autologous (the patient s own) CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence. The concentration is 1 10 106 CD34+ cells/mL.
• The other ingredient is sodium chloride (see section 2, Strimvelis contains sodium ).

What Strimvelis looks like and contents of the pack Strimvelis is a cloudy to clear, colourless to pink dispersion of cells for infusion, which is supplied in one or more infusion bags. The infusion bags are provided in a closed container.

Marketing Authorisation Holder
Fondazione Telethon ETS Via Varese 16/B 00185 Rome Italy

Manufacturer AGC Biologics S.p.A. 58 Via Olgettina 20Milan ITALY

This leaflet was last revised in MM/YYYY.

Other sources of information

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