FDA broadens access to onasemnogene abeparvovec-brve gene therapy for spinal muscular atrophy

The US Food and Drug Administration (FDA) has approved onasemnogene abeparvovec-brve (Itvisma™) for the treatment of spinal muscular atrophy (SMA) in patients aged 2 years and older, including adolescents and adults with confirmed SMN1 gene mutations.

Onasemnogene abeparvovec-brve, a one-time intrathecal gene replacement therapy, expands the pioneering SMA gene-therapy landscape beyond infants, offering a potential one-off treatment that directly targets the underlying genetic defect and may reduce dependence on chronic therapies. This approval represents a landmark shift in the therapeutic options available across the SMA age spectrum.¹

Novartis designed onasemnogene abeparvovec-brve to deliver a functional SMN1 gene via a single fixed intrathecal dose, eliminating the need for weight-based adjustments that are required with earlier formulations. The FDA’s decision draws on robust evidence from registrational studies showing meaningful improvements in motor function and a consistent safety profile across older children and adults.²

Experts and patient groups have widely welcomed the decision. Vinay Prasad, MD, MPH, the FDA’s Chief Medical and Scientific Officer, said the approval underscores “the power of gene therapies” to transform care across diverse SMA populations. Angela Lek, PhD, Chief Research Officer at the Muscular Dystrophy Association, described the milestone as the culmination of decades of collaborative research and advocacy. Kenneth Hobby, President of Cure SMA, highlighted the broader access it offers to families who have long awaited treatment options for older SMA patients.^3,4,5,6

 What 3 things can HCPs learn from this approval?

1. First, the approval decision signals a clear regulatory shift towards broadening access to disease-modifying therapies beyond early childhood, reinforcing the importance of ongoing patient identification, genetic confirmation, and long-term follow-up across the lifespan. For clinicians, this underscores the need to revisit treatment discussions with older children, adolescents, and adults who may previously have been considered ineligible for gene-replacement approaches.
2. Second, the approval highlights the growing acceptance of innovative delivery routes, such as intrathecal administration, to address anatomical and safety limitations seen with earlier systemic gene therapies. This may encourage clinicians to engage more actively in multidisciplinary discussions around procedural pathways, monitoring protocols, and shared decision-making with patients and families.
3. Finally, onasemnogene abeparvovec-brve’s approval reflects the increasing emphasis on functional outcomes and quality-of-life measures in regulatory decision-making.

References

1. 1. FDA approves gene therapy for treatment of spinal muscular atrophy – press announcement. U.S. Food and Drug Administration. 24 Nov 2025. Available at: https://www.fda.gov/news-events/press-announcements/fda-approves-gene-therapy-treatment-spinal-muscular-atrophy (accessed 12 January 2026).
   2. Novartis receives FDA approval for Itvisma®, the first and only gene replacement therapy for children ≥2 years and older with SMA. Novartis press release. Nov 2025. Available at: 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 (accessed 12 January 2026).
   3. FDA approves gene replacement therapy Itvisma for patients with spinal muscular atrophy. Pharmacy Times. Nov 25 2025. Available at: https://www.pharmacytimes.com/view/fda-approves-gene-replacement-therapy-itvisma-for-patients-with-spinal-muscular-atrophy (accessed 12 January 2026).
   4. FDA approves new intrathecal administration route for spinal muscular atrophy gene therapy. NeurologyLive. 2025. Available at: https://www.neurologylive.com/view/fda-approves-new-intrathecal-administration-route-spinal-muscular-atrophy-gene-therapy (accessed 12 January 2026).
   5. Muscular Dystrophy Association calls FDA approval of Itvisma a major step forward for the SMA community. MDA press release. 2025. Available at: https://www.mda.org/press-releases/mda-calls-fda-approval-of-novartis-itvisma-a-major-step-forward (accessed 12 January 2026).
   6. Cure SMA welcomes broad FDA approval of Itvisma for spinal muscular atrophy. PR Newswire. 26 Nov 2025. Available at: https://www.prnewswire.com/news-releases/cure-sma-welcomes-broad-fda-approval-of-itvisma-for-spinal-muscular-atrophy-reinforces-commitment-to-patient-access-and-support-302626958.html (accessed 12 January 2026).