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.