Novartis Zolgensma Gene Therapy Has Significant Long-Term
Zolgensma (onasemnogene abeparvovec), Novartis’ gene therapy for spinal muscular atrophy (SMA), recently announced landmark long-term follow-up data that children treated with Zolgensma were able to maintain key motor functions after 10 years, with some patients even walking independently.
This result further strengthens Zolgensma’s position as a “one-time cure” for SMA and provides a new focus for the global debate on pricing models for high-priced gene therapies.
Novartis’ Zolgensma has become the centre of attention in the latest developments in the medical field. The drug has not only demonstrated significant efficacy in clinical trials but has also been a huge success in the marketplace.
A Breakthrough From Survival To Walking
According to data from the START clinical trial Long-Term Extension Study (LT-001), published in March 2025, 13 of the 15 children with SMA type 1 initially enrolled in the trial completed up to 10 years of follow-up. The results showed:
All children treated with the standard dose survived and did not require permanent ventilator support.
Five children were able to stand with assistance, and two walked independently, a progress that had been considered impossible in SMA type 1 patients.
The safety profile was favorable, with no serious adverse events directly related to treatment, and common complications (e.g., pneumonia) primarily related to the SMA itself.
Zolgensma Clinical Trial Results
The results of clinical trials of Zolgensma have been encouraging. A long-term follow-up study showed that patients treated with Zolgensma showed sustained improvement in motor function over a period of up to 7.5 years with no treatment-related serious adverse events.
In addition, in the SPR1NT trial, all treated presymptomatic children achieved survival without respiratory or nutritional support and sat alone for ≥30 seconds.
Gene Therapy Competitive Landscape And Future Directions
The success of Zolgensma has fueled the rapid development of AAV (adeno-associated virus) vector gene therapy. There are currently more than 200 AAV gene therapies in the R&D stage globally, covering areas such as hemophilia and ophthalmic diseases.
Novartis itself plans to expand the indications for Zolgensma by 2025, further strengthening its market position.
However, challenges remain:
Production complexity leads to high costs.
Competitor threats: the convenience of Roche’s oral drug Evrysdi, for example, and the rise of potential next-generation gene-editing therapies.
Conclusion
Zolgensma’s long-term efficacy data offer hope for SMA patients, but its commercialization pathway still has to deal with multiple tests of pricing, healthcare access and market competition.
As gene therapy technology matures, how to make “sky-high priced therapies” benefit more patients will become a topic for the industry and regulators to explore together.
However, DengYue’s efforts to increase the affordability of the medicine are critical to ensuring that more patients benefit from it.
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