Source: European Medicines Agency (EU) Revision Year: 2020 Publisher: Novartis Gene Therapies EU Limited, Block B, The Crescent Building, Northwood, Santry, Dublin 9, D09 C6X8, Ireland
Zolgensma 2 × 1013 vector genomes/mL solution for infusion.
| Pharmaceutical Form |
|---|
|
Solution for infusion. When thawed, it is a clear to slightly opaque, colourless to faint white solution. |
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.
Each mL contains onasemnogene abeparvovec with a nominal concentration of 2 × 1013 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 (see section 4.4).
For the full list of excipients, see section 6.1.
| Active Ingredient | Description | |
|---|---|---|
| Onasemnogene abeparvovec |
Onasemnogene abeparvovec is a gene therapy designed to introduce a functional copy of the survival motor neuron gene (SMN1) in the transduced cells to address the monogenic root cause of the disease. By providing an alternative source of SMN protein expression in motor neurons, it is expected to promote the survival and function of transduced motor neurons. |
| List of Excipients |
|---|
|
Tromethamine |
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 fill volume sizes, either 5.5 mL or 8.3 mL.
The dose of onasemnogene abeparvovec and exact number of vials required for each patient is calculated according to the patient’s weight (see section 4.2 and Table 5 below).
Table 5. Carton/kit configurations:
| Patient weight (kg) | 5.5 mL viala | 8.3 mL vialb | Total vials per carton |
|---|---|---|---|
| 2.6–3.0 | 0 | 2 | 2 |
| 3.1–3.5 | 2 | 1 | 3 |
| 3.6–4.0 | 1 | 2 | 3 |
| 4.1–4.5 | 0 | 3 | 3 |
| 4.6–5.0 | 2 | 2 | 4 |
| 5.1–5.5 | 1 | 3 | 4 |
| 5.6–6.0 | 0 | 4 | 4 |
| 6.1–6.5 | 2 | 3 | 5 |
| 6.6–7.0 | 1 | 4 | 5 |
| 7.1–7.5 | 0 | 5 | 5 |
| 7.6–8.0 | 2 | 4 | 6 |
| 8.1–8.5 | 1 | 5 | 6 |
| 8.6–9.0 | 0 | 6 | 6 |
| 9.1–9.5 | 2 | 5 | 7 |
| 9.6–10.0 | 1 | 6 | 7 |
| 10.1–10.5 | 0 | 7 | 7 |
| 10.6–11.0 | 2 | 6 | 8 |
| 11.1–11.5 | 1 | 7 | 8 |
| 11.6–12.0 | 0 | 8 | 8 |
| 12.1–12.5 | 2 | 7 | 9 |
| 12.6–13.0 | 1 | 8 | 9 |
| 13.1–13.5 | 0 | 9 | 9 |
| 13.6–14.0 | 2 | 8 | 10 |
| 14.1–14.5 | 1 | 9 | 10 |
| 14.6–15.0 | 0 | 10 | 10 |
| 15.1–15.5 | 2 | 9 | 11 |
| 15.6–16.0 | 1 | 10 | 11 |
| 16.1–16.5 | 0 | 11 | 11 |
| 16.6–17.0 | 2 | 10 | 12 |
| 17.1–17.5 | 1 | 11 | 12 |
| 17.6–18.0 | 0 | 12 | 12 |
| 18.1–18.5 | 2 | 11 | 13 |
| 18.6–19.0 | 1 | 12 | 13 |
| 19.1–19.5 | 0 | 13 | 13 |
| 19.6–20.0 | 2 | 12 | 14 |
| 20.1–20.5 | 1 | 13 | 14 |
| 20.6–21.0 | 0 | 14 | 14 |
a Vial nominal concentration is 2 × 1013 vg/mL and contains an extractable volume of not less than 5.5 mL.
b Vial nominal concentration is 2 × 1013 vg/mL and contains an extractable volume of not less than 8.3 mL.
Novartis Gene Therapies EU Limited, Block B, The Crescent Building, Northwood, Santry, Dublin 9, D09 C6X8, Ireland
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
EU/1/20/1443/013
EU/1/20/1443/014
EU/1/20/1443/015
EU/1/20/1443/016
EU/1/20/1443/017
EU/1/20/1443/018
EU/1/20/1443/019
EU/1/20/1443/020
EU/1/20/1443/021
EU/1/20/1443/022
EU/1/20/1443/023
EU/1/20/1443/024
EU/1/20/1443/025
EU/1/20/1443/026
EU/1/20/1443/027
EU/1/20/1443/028
EU/1/20/1443/029
EU/1/20/1443/030
EU/1/20/1443/031
EU/1/20/1443/032
EU/1/20/1443/033
EU/1/20/1443/034
EU/1/20/1443/035
EU/1/20/1443/036
EU/1/20/1443/037
18 May 2020
| Drug | Countries | |
|---|---|---|
| ZOLGENSMA | Austria, Brazil, Estonia, Finland, France, Croatia, Ireland, Israel, Italy, Lithuania, Poland, Romania, United Kingdom |
© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.
