The gene therapy specifically is indicated for SMA patients with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and three or fewer copies of the survival motor neuron 2 (SMN2) gene or infantile-onset SMA.

Zolgensma replaces the missing or defective SMN1 gene. It is administered by intravenous infusion to deliver the SMN1 gene into a patient's cells and restoring production of the SMN1 protein.

Health Canada's decision is based on efficacy and safety data from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and two copies of the SMN2 gene, as well as patients with presymptomatic genetically diagnosed SMA and two or three copies of the SMN2 gene.

In 2019, the U.S. Food and Drug Administration (FDA) accepted the biologics license application for Zolgensma submitted by AveXis, a biotechnology company that was acquired by Novartis in 2018. AveXis changed its name to Novartis Gene Therapies in September 2020.