Strimvelis
Country: Unjoni Ewropea
Lingwa: Ingliż
Sors: EMA (European Medicines Agency)
Fuljett ta 'informazzjoni
(PIL)
19-09-2023
Karatteristiċi tal-prodott
(SPC)
19-09-2023
Rapport ta 'Valutazzjoni Pubblika
(PAR)
08-06-2016
Ingredjent attiv:
autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells
Disponibbli minn:
Fondazione Telethon ETS
Kodiċi ATC:
L03
INN (Isem Internazzjonali):
autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
Grupp terapewtiku:
Immunostimulants,
Żona terapewtika:
Severe Combined Immunodeficiency
Indikazzjonijiet terapewtiċi:
Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available (see section 4.2 and section 4.4).
Sommarju tal-prodott:
Revision: 9
L-istatus ta 'awtorizzazzjoni:
Authorised
Data ta 'l-awtorizzazzjoni:
2016-05-26
Fuljett ta 'informazzjoni
21
B. PACKAGE LEAFLET
22
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT OR CARER
STRIMVELIS 1–10 × 10
6
CELLS/ML DISPERSION FOR INFUSION
Autologous CD34
+
enriched cell fraction that contains CD34
+
cells transduced with retroviral vector that
encodes for the human ADA cDNA sequence
This medicine is subject to additional monitoring. This will allow
quick identification of new safety
information. You can help by reporting any side effects your child may
get. See the end of section 4 for how
to report side effects.
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOUR CHILD IS GIVEN THIS
MEDICINE BECAUSE IT CONTAINS IMPORTANT
INFORMATION FOR YOUR CHILD.
-
Keep this leaflet. You may need to read it again.
-
If you have any further questions, ask your child’s doctor or nurse.
-
If your child gets any side effects, talk to your child’s doctor or
nurse. This includes any possible side
effects not listed in this leaflet. See section 4.
-
Your child’s doctor will give you a Patient Alert Card which
contains important safety information
about your child’s treatment with Strimvelis. Read it carefully and
follow the instructions on it.
-
Carry the Patient Alert Card with you at all times and always show it
to the doctor or nurse when your
child sees them or if your child goes to hospital.
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
1.
WHAT STRIMVELIS IS AND WHAT IT IS USED FOR
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
(a
_denosine 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 i
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Karatteristiċi tal-prodott
1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
This medicinal product is subject to additional monitoring. This will
allow quick identification of new
safety information. Healthcare professionals are asked to report any
suspected adverse reactions. See section
4.8 for how to report adverse reactions.
1.
NAME OF THE MEDICINAL PRODUCT
Strimvelis 1-10 x 10
6
cells/mL dispersion for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1
GENERAL DESCRIPTION
An autologous CD34
+
enriched cell fraction that contains CD34
+
cells transduced with retroviral vector that
encodes for the human adenosine deaminase (ADA) cDNA sequence from
human haematopoietic
stem/progenitor (CD34
+
) cells.
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
The medicinal product is packaged in one or more infusion bags. Each
patient-specific infusion bag of
Strimvelis contains an autologous CD34
+
enriched cell fraction that contains CD34
+
cells transduced with
retroviral vector that encodes for the human ADA cDNA sequence.
The quantitative information regarding CD34
+
cells/kg and total cells in the product is presented in the
labelling for each batch. The concentration is 1-10 x 10
6
CD34
+
cells/mL.
Excipient with known effect
This medicinal product contains 0.15 mmol sodium per mL.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Dispersion for infusion.
A cloudy to clear, colourless to pink dispersion of cells.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Strimvelis is indicated for the treatment of patients with severe
combined immunodeficiency due to
adenosine deaminase deficiency (ADA-SCID), for whom no suitable human
leukocyte antigen (HLA)-
matched related stem cell donor is available (see sections 4.2 and
4.4).
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Strimvelis must be administered in a specialist transplant centre, by
a physician with previous experience in
the treatment and management of patients with ADA-SCID and in the use
of autologous CD34
+
_ex vivo_
gene
therapy products. It should only be adm
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