ZOLGENSMA Solution for infusion (2020)
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Περιεχόμενα
1. Name of the medicinal product
Zolgensma 2 × 10<sup>13</sup> vector genomes/mL solution for infusion.
2. Qualitative and quantitative composition
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 ...
3. Pharmaceutical form
Solution for infusion. When thawed, it is a clear to slightly opaque, colourless to faint white solution.
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 ...
4.2. Posology and method of administration
Treatment should be initiated and administered in clinical centres and supervised by a physician experienced in the management of patients with SMA. Before administration of onasemnogene abeparvovec, baseline ...
4.3. Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.
4.4. Special warnings and precautions for use
Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded. Pre-existing immunity against AAV9 ...
4.5. Interaction with other medicinal products and other forms of interaction
No interaction studies have been performed. Experience with use of onasemnogene abeparvovec in patients receiving hepatotoxic medication or using hepatotoxic substances is limited. Safety of onasemnogene ...
4.6. Fertility, pregnancy and lactation
Human data on use during pregnancy or lactation are not available and animal fertility or reproduction studies have not been performed.
4.7. Effects on ability to drive and use machines
Onasemnogene abeparvovec has no or negligible influence on the ability to drive and use machines.
4.8. Undesirable effects
Summary of the safety profile The most frequently reported adverse reaction following administration was transient hepatic transaminase increase (12.4%) and vomiting (8.2%), see section 4.4. Tabulated ...
4.9. Overdose
No data from clinical studies are available regarding overdose of onasemnogene abeparvovec. Adjustment of the dose of prednisolone, close clinical observation and monitoring of laboratory parameters (including ...
5.1. Pharmacodynamic properties
<b>Pharmacotherapeutic group:</b> Other drugs for disorders of the musculo-skeletal system <b>ATC code:</b> M09AX09 Mechanism of action Onasemnogene abeparvovec is a gene therapy designed to introduce ...
5.2. Pharmacokinetic properties
Onasemnogene abeparvovec vector shedding studies, which assess the amount of vector eliminated from the body through saliva, urine and faeces were performed. Onasemnogene abeparvovec was detectable in ...
5.3. Preclinical safety data
Following intravenous administration in neonatal mice, vector and transgene were widely distributed with the highest expression generally observed in heart and liver, and substantial expression in the ...
6.1. List of excipients
Tromethamine Magnesium chloride Sodium chloride Poloxamer 188 Hydrochloric acid (for pH adjustment) Water for injections
6.2. Incompatibilities
In the absence of compatibility studies, this medicinal product should not be mixed with other medicinal products.
6.3. Shelf life
1 year. <u>After thawing:</u> Once thawed, the medicinal product should not be re-frozen and may be stored refrigerated at 2°C to 8°C in the original carton for 14 days. Once the dose volume is drawn into ...
6.4. Special precautions for storage
Store and transport frozen (≤ -60°C). Store in a refrigerator (2°C to 8°C) immediately upon receipt. Store in the original carton. For storage conditions after thawing of the medicinal product, see section ...
6.5. Nature and contents of container
Onasemnogene abeparvovec is supplied in a vial (10 mL polymer crystal zenith) with stopper (20 mm chlorobutyl rubber) and seal (aluminum, flip-off) with a coloured cap (plastic), in two different vial ...
6.6. Special precautions for disposal and other handling
This medicinal product contains genetically-modified organisms. Appropriate precautions for the handling, disposal or accidental exposure of onasemnogene abeparvovec should be followed: The onasemnogene ...
7. Marketing authorization holder
Novartis Gene Therapies EU Limited, Block B, The Crescent Building, Northwood, Santry, Dublin 9, D09 C6X8, Ireland
8. Marketing authorization number(s)
EU/1/20/1443/001 EU/1/20/1443/002 EU/1/20/1443/003 EU/1/20/1443/004 EU/1/20/1443/005 EU/1/20/1443/006 EU/1/20/1443/007 EU/1/20/1443/008 EU/1/20/1443/009 EU/1/20/1443/010 EU/1/20/1443/011 EU/1/20/1443/012 ...
9. Date of first authorization / renewal of the authorization
18 May 2020
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