Novartis Prepares to File Intrathecal Zolgensma for Older SMA Patients by Mid-2025

Novartis plans to file for regulatory approval of intrathecal Zolgensma in the first half of 2025 based on positive Phase 3 data13
The intrathecal version is delivered directly into the spinal fluid, allowing it to be used in older and heavier SMA patients compared to the IV formulation13
In the Phase 3 STEER trial, intrathecal Zolgensma improved motor function scores by 2.39 points compared to 0.51 points for placebo in patients aged 2-18 years110
The STRENGTH trial showed stabilization of motor function in patients who switched from other SMA therapies to intrathecal Zolgensma110
Intrathecal delivery uses a much lower dose than IV administration, potentially improving safety for older patients1
If approved, it would expand Zolgensma treatment to SMA patients over 2 years old, a population currently not eligible for the IV version34
Novartis estimates intrathecal Zolgensma could reach multibillion-dollar peak sales if approved for older patients1

Sources:

1. https://www.fiercepharma.com/pharma/novartis-details-intrathecal-zolgensma-data-gene-therapy-bid-reach-more-sma-patients

3. https://www.fiercepharma.com/pharma/after-delay-fda-novartis-touts-pivotal-trial-win-intrathecal-zolgensma-older-sma-patients

4. https://www.biospace.com/drug-development/Novartis-intrathecal-zolgensma-effective-in-older-children

10. https://www.cgtlive.com/view/novartis-oav101-improves-stabilizes-motor-function-patients-spinal-muscular-atrophy