Source: European Medicines Agency (EU) Revision Year: 2026 Publisher: Novartis Europharm Limited, Vista Building, Elm Park, Merrion Road, Dublin 4, Ireland
Zolgensma 2 × 1013 vector genomes/mL solution for infusion.
| Pharmaceutical Form |
|---|
|
Solution for infusion. 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.
For the full list of excipients, see section 6.1.
| Active Ingredient |
|---|
|
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 6 below).
Table 6. 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 Europharm Limited, Vista Building, Elm Park, Merrion Road, Dublin 4, Ireland
EU/1/20/1443/001
EU/1/20/1443/002
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Date of first authorisation: 18 May 2020
Date of latest renewal: 17 May 2022
| Drug | Countries | |
|---|---|---|
| ZOLGENSMA | Austria, Brazil, Estonia, Finland, France, Croatia, Ireland, Israel, Italy, Lithuania, Poland, Romania, United Kingdom |
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