Zolgensma

Maa: Euroopan unioni

Kieli: englanti

Lähde: EMA (European Medicines Agency)

Aktiivinen ainesosa:

onasemnogene abeparvovec

Saatavilla:

Novartis Europharm Limited 

ATC-koodi:

M09AX09

INN (Kansainvälinen yleisnimi):

onasemnogene abeparvovec

Terapeuttinen ryhmä:

Other drugs for disorders of the musculo-skeletal system

Terapeuttinen alue:

Muscular Atrophy, Spinal

Käyttöaiheet:

Zolgensma is indicated for the treatment of:patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, orpatients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

Tuoteyhteenveto:

Revision: 13

Valtuutuksen tilan:

Authorised

Valtuutus päivämäärä:

2020-05-18

Pakkausseloste

38
B. PACKAGE LEAFLET
39
Package leaflet: Information for the user
ZOLGENSMA 2
× 10
13 VECTOR GENOMES/ML SOLUTION FOR INFUSION
onasemnogene abeparvovec
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.
-
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.
WHAT IS IN THIS LEAFLET
1.
What Zolgensma is and what it is used for
2.
What you need to know before your child is given Zolgensma
3.
How Zolgensma is given
4.
Possible side effects
5.
How to store Zolgensma
6.
Contents of the pack and other information
1.
WHAT ZOLGENSMA IS AND WHAT IT IS USED FOR
WHAT ZOLGENSMA IS
Zolgensma is a type of medicine called a ‘gene therapy’. It
contains the active substance
onasemnogene abeparvovec, which contains human genetic material.
WHAT ZOLGENSMA IS USED FOR
Zolgensma is used to treat spinal muscular atrophy (SMA), a rare,
serious inherited disease.
HOW ZOLGENSMA WORKS
SMA occurs when there is a missing or abnormal version of a gene
needed to make an essential
protein called ‘Survival Motor Neuron’ (SMN) protein. Lack of SMN
protein causes nerves that
control muscles (motor neurons) to die. This results in muscles
becoming weak and wasting away,
with eventual loss of movement.
This medicine works by supplying a fully functioning copy of the SMN
gene which then helps the
body produce enough SMN protein. The gene is delivered into the cells
where it is needed using a
modified virus that does not cause disease in humans.
40
2.
WHAT YOU NEED TO KNOW BEFORE YOUR CHILD IS GIVEN ZOLGENSMA
DO NOT US

Valmisteyhteenveto

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
Zolgensma 2 × 10
13
vector genomes/mL solution for infusion
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
2.1
GENERAL DESCRIPTION
Onasemnogene abeparvovec is a gene therapy medicinal product that
expresses the human survival
motor neuron (SMN) protein. It is a non-replicating recombinant
adeno-associated virus serotype 9
(AAV9) based vector containing the cDNA of the human SMN gene under
the control of the
cytomegalovirus enhancer/chicken-β-actin-hybrid promoter.
Onasemnogene abeparvovec is produced in human embryonic kidney cells
by recombinant DNA
technology.
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each mL contains onasemnogene abeparvovec with a nominal concentration
of 2 × 10
13
vector
genomes (vg). Vials will contain an extractable volume of not less
than either 5.5 mL or 8.3 mL. The
total number of vials and combination of fill volumes in each finished
pack will be customised to meet
dosing requirements for individual patients depending on their weight
(see sections 4.2 and 6.5).
Excipient with known effect
This medicinal product contains 0.2 mmol sodium per mL.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Solution for infusion.
A clear to slightly opaque, colourless to faint white solution.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Zolgensma is indicated for the treatment of:
-
patients with 5q spinal muscular atrophy (SMA) with a bi-allelic
mutation in the
_SMN1_
gene
and a clinical diagnosis of SMA Type 1, or
-
patients with 5q SMA with a bi-allelic mutation in the
_SMN1_
gene and up to 3 copies of the
_SMN2_
gene.
3
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Treatment should be initiated and administered in clinical centres and
supe