Product Dossier
ZOLGENSMA
Product Dossier for ZOLGENSMA (onasemnogene abeparvovec, Novartis Pharmaceuticals). ARTG record, PBS listing, PBAC outcomes, AusPAR — compiled by…
- Sponsor: Novartis Pharmaceuticals
- Active ingredient: onasemnogene abeparvovec
- Therapeutic area: Rare Disease
- Same area: ORKAMBI
- Same area: BYLVAY
What it is
Zolgensma is onasemnogene abeparvovec 2 x e13 vg/mL injection for intravenous infusion, sponsored by Novartis Pharmaceuticals.
Approved indications
— Spinal muscular atrophy (SMA) in patients aged less than 9 months, with Type 1 SMA or pre-symptomatic patients with 1-2 copies of the SMN2 gene. — Pre-symptomatic spinal muscular atrophy (SMA).
PBS listing
Zolgensma solution for intravenous infusion 20 trillion vector genomes per mL is listed on the PBS with authority required restriction and an ex-manufacturer price of A$2,527,773.87. The listing includes packs containing 2 vials of 8.3 mL, and multiple other pack configurations are available.
Regulatory history
Zolgensma was first listed on the ARTG on 4 March 2021. In May 2021, the PBAC deferred its recommendation due to concerns with the subsidy proposal, noting that the request for inclusion of patients with 3 copies of SMN2 was not adequately supported and that at the requested price, onasemnogene abeparvovec was unlikely to be cost-effective. In September 2021, the PBAC recommended Zolgensma for spinal muscular atrophy in patients aged less than 9 months with Type 1 SMA or pre-symptomatic patients with 1-2 copies of the SMN2 gene. In November 2022, the PBAC did not recommend extension to presymptomatic treatment of children with 3 copies of the SMN2 gene. In July 2023, the PBAC recommended Zolgensma for pre-symptomatic spinal muscular atrophy.