FDA Approves Novartis’s Itvisma: First Gene Therapy for SMA Patients 2+

On November 24, 2025, the U.S. Food and Drug Administration (FDA) granted approval to Itvisma® (onasemnogene abeparvovec-brve). This treatment is specifically designed for individuals aged two and older who have spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 (SMN1) gene. Itvisma is notable as the first gene replacement therapy available for this demographic.

Significance of Itvisma Approval

Itvisma introduces a one-time treatment option that aims to replace the missing SMN1 gene. This therapy has the potential to improve motor function and reduce the reliance on chronic treatments typically associated with SMA therapies. The approval is a significant milestone, expanding treatment options for children, teens, and adults suffering from this condition.

• First and Only Gene Replacement Therapy: Targets SMA in a broader age group.
• One-Time Dose: A fixed dosage is administered without adjustment for age or weight.
• Improved Motor Function: Evidence suggests notable enhancements in motor capabilities.

Clinical Study Outcomes

The FDA’s decision was informed by two pivotal studies: the Phase III STEER study and the Phase IIIb STRENGTH study. These studies demonstrated:

• Statistically significant motor function improvements.
• Sustained results over a follow-up period of 52 weeks.
• Consistent safety profile with manageable adverse events.

Common adverse reactions in the STEER study included upper respiratory infections and fevers, while the STRENGTH study noted colds and vomiting as prevalent side effects.

Impact on the SMA Community

Experts highlight the transformational potential of Itvisma for SMA patients. Dr. John W. Day from Stanford University described the approval as a breakthrough for patients of all ages. Additionally, Kenneth Hobby from Cure SMA emphasized that this therapy may enhance patient independence in daily activities.

SMA results from a deficiency in the SMN1 gene, essential for muscle function like breathing and basic movement. Approximately 9,000 people in the U.S. live with this rare genetic disorder, which can lead to severe muscle weakness.

Future Prospects

Itvisma will be available in the U.S. starting in December 2025. Novartis is committed to supporting eligible patients through its patient assistance programs. Interested patients or healthcare providers can contact Novartis Patient Support for help with treatment initiation and insurance coverage inquiries.

Victor Bultó, President of Novartis US, articulated this achievement as a pathway to addressing the unmet needs of a broader SMA population. He indicated that Itvisma represents significant progress in innovative treatments for SMA.

In conclusion, Itvisma’s FDA approval marks a new chapter for patients and families affected by SMA, enhancing access to effective genetic treatments for this debilitating condition.