Skysona

Krajina: Európska únia

Jazyk: angličtina

Zdroj: EMA (European Medicines Agency)

Aktívna zložka:

elivaldogene autotemcel

Dostupné z:

bluebird bio (Netherlands) B.V.

ATC kód:

N07

INN (Medzinárodný Name):

elivaldogene autotemcel

Terapeutické skupiny:

Other nervous system drugs

Terapeutické oblasti:

Adrenoleukodystrophy

Terapeutické indikácie:

Treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA) matched sibling haematopoietic stem cell donor is not available.

Stav Autorizácia:

Withdrawn

Dátum Autorizácia:

2021-07-16

Príbalový leták

35
B. PACKAGE LEAFLET
Medicinal product no longer authorised
36
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT OR CARER
SKYSONA 2-30 × 10
6 CELLS/ML DISPERSION FOR INFUSION
elivaldogene autotemcel (autologous CD34
+
cells encoding
_ABCD1_
gene)
This medicine is subject to additional monitoring. This will allow
quick identification of new safety
information. You can help by reporting any side effects you or your
child may get. See the end of
section 4 for how to report side effects.
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU/YOUR CHILD IS GIVEN THIS
MEDICINE BECAUSE IT CONTAINS
IMPORTANT INFORMATION FOR YOU.
-
Keep this leaflet. You may need to read it again.
-
If you have any further questions, ask the doctor or nurse.
-
If you get any side effects, talk to the doctor or nurse. This
includes any possible side effects not
listed in this leaflet. See section 4.
-
The doctor or nurse will give you a Patient Alert Card which contains
important safety information
about the treatment with Skysona. Read it carefully and follow the
instructions on it.
-
Carry the Patient Alert Card with you at all times and always show it
to any doctor or nurse who sees
you or if you go to the hospital.
WHAT IS IN THIS LEAFLET
1.
What Skysona is and what it is used for
2.
What you need to know before you or your child is given Skysona
3.
How Skysona is made and given
4.
Possible side effects
5.
How to store Skysona
6.
Contents of the pack and other information
1.
WHAT SKYSONA IS AND WHAT IT IS USED FOR
Skysona is used to treat a serious genetic disease called cerebral
adrenoleukodystrophy (CALD) in
children and adolescents younger than 18 years old.
People with CALD have an alteration in the gene that makes a protein
called adrenoleukodystrophy
protein (ALDP). People with CALD either cannot make this protein or
have a protein that does not work
well. This leads to the build-up of very long chain fatty acids in the
body, particularly in the brain. These
fatty acids cause severe damage to brain cells. If untreated, this
damage will lead

Súhrn charakteristických

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
Medicinal product no longer authorised
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
Skysona 2-30 × 10
6
cells/mL dispersion for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1
GENERAL DESCRIPTION
Skysona (elivaldogene autotemcel) is a genetically modified autologous
CD34
+
cell-enriched population
that contains haematopoietic stem cells (HSCs) transduced
_ex vivo_
with lentiviral vector (LVV) encoding
_ABCD1 _
complementary deoxyribonucleic acid (cDNA) for human
adrenoleukodystrophy protein
(ALDP).
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each patient-specific infusion bag of Skysona contains elivaldogene
autotemcel at a batch-dependent
concentration of genetically modified autologous CD34
+
cell enriched population. The finished product is
packaged in one or more infusion bags, containing a dispersion of 2-30
× 10
6
cells/mL of CD34
+
enriched
cell population suspended in cryopreservative solution. Each infusion
bag contains approximately 20 mL
of dispersion for infusion.
The quantitative information of medicinal product regarding strength,
CD34
+
cells, and dose for the
specific patient is provided in the Lot Information Sheet. The Lot
Information Sheet is included inside the
lid of the cryoshipper used to transport Skysona.
Excipient(s) with known effect
Each dose contains 391-1564 mg sodium (included in Cryostor CS5).
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Dispersion for infusion.
A colourless to white to red, including shades of white or pink, light
yellow, and orange dispersion.
Medicinal product no longer authorised
3
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Skysona is indicated for the treatment of early cerebral
adrenoleukodystrophy in patients less than
18 yea