Šalis: Izraelis
kalba: anglų
Šaltinis: Ministry of Health
ONASEMNOGENE ABEPARVOVEC
NOVARTIS ISRAEL LTD
M09AX09
SUSPENSION
ONASEMNOGENE ABEPARVOVEC 2 x 10^13 VG / 1 ML
I.V
Required
NOVARTIS GENE THERAPIES, INC, USA
ONASEMNOGENE ABEPARVOVEC
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.
2020-11-02
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 Perskaitykite visą dokumentą
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 Perskaitykite visą dokumentą