Glybera
Land: Den Europæiske Union
Sprog: engelsk
Kilde: EMA (European Medicines Agency)
Indlægsseddel
(PIL)
10-07-2017
Produktets egenskaber
(SPC)
10-07-2017
Offentlige vurderingsrapport
(PAR)
15-06-2016
Aktiv bestanddel:
alipogene tiparvovec
Tilgængelig fra:
uniQure biopharma B.V.
ATC-kode:
C10AX10
INN (International Name):
alipogene tiparvovec
Terapeutisk gruppe:
Lipid modifying agents
Terapeutisk område:
Hyperlipoproteinemia Type I
Terapeutiske indikationer:
Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein.
Produkt oversigt:
Revision: 8
Autorisation status:
Withdrawn
Autorisation dato:
2012-10-25
Indlægsseddel
26
B.
PACKAGE LEAFLET
Medicinal product no longer authorised
27
PACKAGE LEAFLET: INFORMATION FOR THE USER
GLYBERA 3 X 10
12 GENOME COPIES /ML SOLUTION FOR INJECTION
Alipogene tiparvovec
▼ 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 may
get. See the end of section 4
for how to report side effects
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU ARE 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 your doctor.
-
If you get any side effects, talk to your doctor. This includes any
possible side effect not listed
in the leaflet.
-
You have been given a patient card by your doctor. Read it carefully
and follow the related
instructions.
-
You should present this card to your health care professionals
(doctor, nurse) upon
consultation or hospitalisation. See section 4
WHAT IS IN THIS LEAFLET
:
1.
What Glybera is and what it is used for
2.
What you need to know before you are given Glybera
3.
How Glybera is given to you
4.
Possible side effects
5.
How to store Glybera
6.
Contents of the pack and other information
1.
WHAT GLYBERA IS AND WHAT IT IS USED FOR
Glybera contains alipogene tiparvovec, a gene therapy product that
works by delivering a gene into the
body to correct a genetic deficiency. It belongs to a group of
medicines called lipid modifying agents.
Glybera is used to treat a specific inherited condition known as
“lipoprotein lipase deficiency
(LPLD)”.
Lipoprotein lipase (LPL) is a naturally occurring substance in the
body (known as an enzyme) that
controls the level of certain fats in the blood. In lipoprotein lipase
deficiency, this enzyme is missing
due to a genetic defect. People who are affected by this condition
have a build up of very high fat
levels in their blood (hyperchylomicronemia).
Glybera is used to treat adult patients diagnosed with lipoprotein
lipase deficiency (L
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Produktets egenskaber
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
Glybera 3 × 10
12
genome copies/ml solution for injection
2. QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1 GENERAL DESCRIPTION
Alipogene tiparvovec contains the human lipoprotein lipase (LPL) gene
variant LPL
S447X
in a vector.
The vector comprises a protein shell derived from adeno-associated
virus serotype 1 (AAV1), the
Cytomegalovirus (CMV) promoter, a woodchuck hepatitis virus
posttranscriptional regulatory element
and AAV2 derived inverted terminal repeats. Alipogene tiparvovec is
produced using insect cells and
recombinant baculovirus technology.
2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION
Each vial of alipogene tiparvovec contains 1 extractable ml of
solution, containing 3 x 10
12
genome
copies (gc).
Each patient-specific pack contains a sufficient amount of vials to
dose each patient with 1 x 10
12
LPL
S447X
gc/kg bodyweight.
Excipient with known effect:
This medicinal product contains 47.5 mg sodium per administration at
27 injection sites to 105.6 mg
sodium per administration at 60 injection sites.
For the full list of excipients, see section 6.1.
3. PHARMACEUTICAL FORM
Solution for injection.
Clear, to slightly opalescent, colourless solution.
4. CLINICAL PARTICULARS
4.1 THERAPEUTIC INDICATIONS
Glybera is indicated for adult patients diagnosed with familial
lipoprotein lipase deficiency (LPLD)
and suffering from severe or multiple pancreatitis attacks despite
dietary fat restrictions
.
The diagnosis
of LPLD has to be confirmed by genetic testing. The indication is
restricted to patients with detectable
levels of LPL protein (see section 4.4).
4.2 POSOLOGY AND METHOD OF ADMINISTRATION
Glybera should only be used when the diagnosis
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