RegulatoryMar 30, 2026, 03:07 AM
Novartis Receives FDA Approval for Itvisma Gene Therapy for Spinal Muscular Atrophy (SMA) for Broad Age Range
AI Summary
Novartis announced that the FDA has approved Itvisma (onasemnogene abeparvovec-brve), a gene replacement therapy, for the treatment of spinal muscular atrophy (SMA) in patients two years and older, including teens and adults. This approval expands the availability of gene therapy to a broader SMA patient population, potentially reducing the need for chronic treatments.
Key Highlights
- Itvisma is the first and only gene replacement therapy approved for a broad age range of SMA patients.
- The approval is based on Phase III STEER and STRENGTH study data demonstrating improved motor function and stabilization.
- Itvisma is administered as a one-time dose, replacing the SMN1 gene to address the genetic root cause of SMA.
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