The FDA has approved Isembyld as the first spinal muscular atrophy treatment designed to directly target muscle loss, for patients aged 2 years and older receiving an SMN2-targeted therapy.

The decision makes Itvisma the first and only gene replacement therapy approved in the EU for older children, teenagers and adults living with 5q spinal muscular atrophy. The European Commission has approved Novartis' Itvisma (onasemnogene abeparvovec) for the treatment of older children, teenagers and adults with 5q spinal muscular atrophy (SMA) who have a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene. The approval significantly expands access to gene replac