Shots:
- The US FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of pts (≥2yrs.) living with spinal muscular atrophy (SMA) harboring a confirmed mutation in the SMN1 gene; US availability expected in Dec 2025
- Approval was based on the P-III (STEER) trial & open-label P-IIIb (STRENGTH) study, showing Itvisma improved motor function & stabilized motor abilities, with effects sustained over 52wks. of follow-up; results were presented at 2025 MDA Clinical & Scientific Conference
- Itvisma is a gene replacement therapy that delivers a functional SMN1 gene via a single intrathecal dose, enabling sustained SMN protein expression to improve motor function in SMA pts
Ref: Novartis | Image: Novartis | Press Release
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