Novartis recently announced new anticipated long-term positive data for its one-time infusion gene therapy, Zolgensma (onasemnogene abeparvovec-xioi), which is set to help the drug generate global sales of $2.5bn by 2025 and lead the spinal muscular atrophy (SMA) market, says GlobalData, a leading data and analytics company.

The data showed significant therapeutic benefit in patients with SMA over a range of extended studies, covering patients treated presymptomatically, and sustained durability in patients up to five years post-dosing.
Interim results were also reported of an intrathecal formulation testing in older patients with SMA type 2 aged two to five.

Sarah Elsayed, Pharma Analyst at GlobalData, commented: “The robust overall trial results are expected to spur the drug’s sales, expand its future patient pool and further dispel controversies raised over the drug’s credibility after its preclinical data manipulation incident and $2.1m price tag.’’

The unveiled impressive data will give Novartis a competitive advantage over other marketed SMA therapies, posing a threat to Biogen’s blockbuster Spinraza (nusinersen). Zolgensma, despite costing $2.1m per infusion, is thought to be more cost-effective than Spinraza over the long term.

Elsayed continued, “Given Zolgensma’s five-year efficacy and safety results, coupled with the drug’s approval in Japan and the findings from a new study investigating expanded use in patients with SMA type 2, GlobalData expects that Zolgensma will change the landscape of the SMA market and it will prove that gene therapies may hold the key to SMA treatment.”

The presented data of a completed pivotal STR1VE-US study showed that nearly all (91%) patients met the co-primary efficacy endpoint of event-free survival at 14 months, and more than half (59%) of patients met the co-primary efficacy endpoint of sitting for ≥30 seconds at 18 months of age – a milestone never achieved before in any clinical trial of SMA Type 1. Additionally, nine of 22 patients demonstrated the ‘ability to thrive’ at 18 months of age.