Zolgensma Description

A clear to slightly opaque, colourless to faint white solution.
Each mL contains onasemnogene abeparvovec with a nominal concentration of 2 x 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 Nature and contents of container as follows and Dosage & Administration).
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.
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 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 Table 1 as follows and Dosage & Administration).

Click on icon to see table/diagram/image

Excipient with known effect: This medicinal product contains 0.2 mmol sodium per mL.
Excipients/Inactive Ingredients: Tromethamine, Magnesium chloride, Sodium chloride, Poloxamer 188, Hydrochloric acid (for pH adjustment), Water for injections.