FDA Approves Novartis’ Itvisma: Expanding Gene Therapy to Older Spinal Muscular Atrophy Patients
The FDA has approved Itvisma (onasemnogene abeparvovec-brve), a new intrathecal formulation of Novartis' gene therapy, for the treatment of children two years and older, teens, and adults with spinal muscular atrophy (SMA) who have a confirmed SMN1 gene mutation157317.
This approval makes Itvisma the first and only gene-replacement therapy available to a broader SMA population beyond infants, including older children and adults1531317.
Itvisma's expanded use is enabled by the intrathecal (spinal) delivery method, allowing safe and effective treatment in patients who are too old or too large for the original intravenous (IV) Zolgensma formulation24712.
Clinical trials, including the Phase 3 STEER study, demonstrated statistically significant improvements in motor function and stabilization of abilities over 52 weeks in older patients, supporting the FDA's decision524.
Prior to this, Zolgensma was only approved for children less than two years old and delivered intravenously; Itvisma now covers nearly the entire SMA patient population, fulfilling a longstanding unmet medical need181012.
The Muscular Dystrophy Association (MDA) and other advocacy organizations have praised the approval as a major advance for the SMA community, offering new hope for patients and families316.
Safety labeling for Itvisma closely follows that of Zolgensma, retaining boxed warnings for hepatotoxicity and other risks identified in real-world use of the IV formulation7.
Novartis plans to launch Itvisma in the US in December 2025, potentially expanding access to approximately 9,000 Americans living with SMA1.
Sources:
1. https://www.finanzen.ch/nachrichten/aktien/novartis-wins-fda-nod-for-gene-replacement-therapy-itvisma-to-treat-spinal-muscular-atrophy-1035593371
2. https://www.fiercepharma.com/pharma/novartis-details-intrathecal-zolgensma-data-gene-therapy-bid-reach-more-sma-patients
3. https://www.mda.org/press-releases/mda-calls-fda-approval-of-novartis-itvisma-a-major-step-forward
4. https://www.novartis.com/news/media-releases/novartis-presents-new-data-safety-and-efficacy-zolgensma-including-maintained-and-improved-motor-milestones-older-and-heavier-children-sma
5. https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-itvisma-only-gene-replacement-therapy-children-two-years-and-older-teens-and-adults-spinal-muscular-atrophy-sma
7. https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy
8. https://www.statnews.com/2025/03/19/spinal-muscular-atrophy-novartis-zolgensma-older-children/
10. https://www.novartis.com/news/media-releases/avexis-statement-access-zolgensma-onasemnogene-abeparvovec-xioi
12. https://pharmatimes.com/news/novartis-gene-therapy-set-to-enhance-sma-treatment/
13. https://pharmaphorum.com/news/novartis-sma-gene-therapy-cleared-wider-use-us
16. https://smanewstoday.com/news/mda-2025-sma-gene-therapy-transformational-novartis-exec/
17. https://www.thepharmaletter.com/biotechnology/novartis-scores-a-first-with-fda-for-itvisma