ZOLGENSMA

Maa: Israel

Kieli: englanti

Lähde: Ministry of Health

Aktiivinen ainesosa:

ONASEMNOGENE ABEPARVOVEC

Saatavilla:

NOVARTIS ISRAEL LTD

ATC-koodi:

M09AX09

Lääkemuoto:

SUSPENSION

Koostumus:

ONASEMNOGENE ABEPARVOVEC 2 x 10^13 VG / 1 ML

Antoreitti:

I.V

Prescription tyyppi:

Required

Valmistaja:

NOVARTIS GENE THERAPIES, INC, USA

Terapeuttinen alue:

ONASEMNOGENE ABEPARVOVEC

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, or- patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

Valtuutus päivämäärä:

2020-11-02

Pakkausseloste

Your child’s doctor has given you this guide because your child
has been prescribed Zolgensma.
This guide aims to provide practical information to support
discussions with your doctor. It should be read along with the
Patient information leaflet you receive for this medicine
If your child experiences any side effects, talk to your child’s
doctor or nurse. This includes any possible side effects not
listed in this guide or the Patient information leaflet delivered
together with this guide
IF YOU HAVE ANY QUESTIONS OR CONCERNS ABOUT ZOLGENSMA
SPEAK WITH YOUR CHILD’S DOCTOR OR NURSE.
ZOLGENSMA®
PARENT’S /
CAREGIVER GUIDE
RMP 2023-005-ENG
WHAT IS IN THIS GUIDE?
THIS GUIDE HAS BEEN DEVELOPED TO PROVIDE USEFUL INFORMATION ON:
• ZOLGENSMA AND HOW IT WORKS
• IMPORTANT SAFETY INFORMATION AND POSSIBLE SIDE EFFECTS OF
ZOLGENSMA
• EACH STEP OF THE ZOLGENSMA TREATMENT JOURNEY
If you have any questions or concerns about this medicine or your
child’s
health and wellbeing please speak with your child’s doctor or nurse.
ABOUT SMA
•
What is SMA?
•
What causes SMA?
•
Who gets SMA?
•
Severity and symptoms of SMA
4
4
6
8
10
ABOUT ZOLGENSMA
Learn about Zolgensma and how it works
12
UNDERSTANDING THE RISKS OF ZOLGENSMA
Important safety information and possible side effects of Zolgensma
14
TREATMENT WITH ZOLGENSMA
•
Before treatment
•
Treatment day
•
After treatment
19
20
22
23
WHEN TO SEEK MEDICAL ATTENTION
29
PHYSICIAN CONTACT DETAILS
30
LOCAL PATIENTS ASSOCIATIONS
31
COMMON WORDS TO KNOW
Find helpful definitions for terms used in this guide
32
SMA, spinal muscular atrophy.
SMA, spinal muscular atrophy.
USEFUL CONTACTS
RMP 2023-005-ENG
ABOUT SMA | 5
ABOUT SMA
WHAT IS SMA?
SMA, spinal muscular atrophy; SMN, survival motor neuron.
SMA, spinal muscular atrophy.
HEALTHY PEOPLE
PEOPLE AFFECTED BY SMA
SPINAL MUSCULAR ATROPHY (SMA) is a rare, serious, inherited condition.
SMA occurs when
there is a missing or abnormal version of a gene needed to make an
essential protein
called ‘Survival motor neuron’ (SM

Valmisteyhteenveto

ZOL API NOV23 V4
1
EU SmPC Oct2023
PRODUCT INFORMATION
1.
NAME OF THE MEDICINAL PRODUCT
ZOLGENSMA PARENT'S/CAREGIVER SAFETY INFORMATION GUIDE
The marketing of Zolgensma is subject to a risk management plan (RMP)
including a
`Parent's/Caregiver safety information guide`. The `Parent's/Caregiver
safety information guide`
emphasizes important safety information that the parents/caregivers
should be aware of before and
during treatment.
Please explain to the parents/caregivers the need to review the card
before starting treatment.
HEALTH CARE PROVIDERS (HCP) GUIDE
This product is marketed with an HCP guide providing important safety
information. Please ensure
you are familiar with this material as it contains important safety
information.
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.
WARNING: SERIOUS LIVER INJURY AND ACUTE LIVER FAILURE
•
Cases of acute liver failure with fatal outcomes have been reported.
Acute serious liver injury
and elevated aminotransferases can also occur with onasemnogene
abeparvovec (see section 4.4).
•
Patients with preexisting liver impairment may be at higher risk (see
sections 4.2 and 4.4).
•
Prior to infusion, assess liver function of all patients by clinical
examination and laboratory
testing. Administer systemic corticosteroid to all patients before and
after onasemnogene
abeparvovec infusion. Continue to monitor liver function for at least
3 months after infusion, and
at other times as clinically indicated (see sections 4.2 and 4.4).
ZOL API NOV23 V4
2
EU SmPC Oct2023
2.2
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each mL con