Shots:
- The CHMP has recommended Novartis‘ Itvisma (onasemnogene abeparvovec) for the treatment of pts (≥2yrs.) living with 5q spinal muscular atrophy (SMA) harboring a confirmed bi-allelic mutation in the SMN1 gene
- Opinion was based on the P-III (STEER) trial & P-IIIb (STRENGTH) study, where STEER showed a 2.39-point improvement in the HFMSE with effects sustained over 52wks. vs 0.51 in Sham. Both trials demonstrated benefit in treatment-naïve as well as experience pts
- 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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