Novartis Wins European Approval For Spinal Muscular Atrophy Gene Therapy
MT Newswires Live
Jul 02
Novartis (NVS) has received European Commission approval for its gene replacement treatment Itvisma to treat spinal muscular atrophy in patients aged 2 and older.
The single-dose therapy works by substituting the defective SMN1 gene to improve physical movement without requiring weight-based or age-based changes, the company said.
Regulatory clearance depended primarily on data from a trial where patients demonstrated improved motor function over a 52-week observation span, Novartis said.
Shares of the company were up 1.7% in Thursday premarket activity.
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