FDA Grants Priority Review For Evrysdi To Treat Pre-symptomatic Babies With Spinal Muscular Atrophy

PTC Therapeutics Inc. (PTCT) said on Tuesday that its supplemental New Drug Application seeking to expand the label of Evrysdi to include pre-symptomatic infants under 2 months old with spinal muscular atrophy has been accepted for priority review by the FDA.

Spinal muscular atrophy is a severe, progressive neuromuscular disease caused by a mutation of the survival motor neuron 1 (SMN1) gene, leading to a deficiency of SMN protein that is essential to the function of nerves that control muscles and movement.

Evrysdi received FDA approval in August 2020 for the treatment of spinal muscular atrophy for adults and children 2 months and older. The drug is administered daily at home in liquid form by mouth or feeding tube.

If approved for the proposed indication, Evrysdi would be the first medicine administered at-home for pre-symptomatic babies with spinal muscular atrophy.

Evrysdi is developed as part of collaboration between Roche, PTC Therapeutics and the SMA Foundation.

In the nine months ended September 30, 2021, Evrysdi generated sales of CHF 396 million for Roche, resulting in $33.3 million in year-to-date royalties to PTC Therapeutics.

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