Evrysdi is granted FDA priority review for treatment of pre-symptomatic babies under 2 months of age with spinal muscular atrophy.

The sNDA submission incorporates interim data from the RAINBOWFISH study, which shows the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking and maintained the ability to swallow following 12 months of treatment.

Evrysdi is designed to treat SMA by increasing and sustaining production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi’s existing FDA label is for the treatment of SMA in adults, children and babies two months and older. If approved, Evrysdi would be the first medicine administered at home for pre-symptomatic babies with SMA.