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Zolgensma
Published
Product name
Zolgensma
Active ingredient
Onasemnogene abeparvovec
Submission type
New biological entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Zolgensma (onasemnogene abeparvovec) was approved for the following therapeutic use:
Zolgensma (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 1 to 3 copies of the SMN2 gene.
How this medicine works
Spinal muscular atrophy (SMA) is caused by a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene, which results in insufficient survival motor neuron (SMN) protein expression. Zolgensma (onasemnogene abeparvovec) is a non-replicating recombinant adeno-associated viral (AAV) vector that utilises the AAV9 capsid to deliver a stable fully functional copy of the transgene encoding the human survival motor gene protein. The SMN1 gene present in onasemnogene abeparvovec is designed to reside as episomal DNA in the nucleus of transduced cells and is expected to be stably expressed for an extended period of time in post-mitotic cells. The AAV9 virus is not known to cause disease in humans. Intravenous administration of Zolgensma that results in cell transduction and expression of the SMN protein has been observed in two human case studies (see Section 5.2 Pharmacokinetic properties in the Product Information).
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Zolgensma was considered favourable for the therapeutic use approved.
ARTG details
- ZOLGENSMA onasemnogene abeparvovec 2 x e13 vg/mL injection for intravenous infusion 8.3 mL vial (327905)
- ZOLGENSMA onasemnogene abeparvovec 2 x e13 vg/mL injection for intravenous infusion 5.5 mL vial (327906)
- ZOLGENSMA onasemnogene abeparvovec 2 x e13 vg/mL injection for intravenous infusion pack (327907)