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PTC Therapeutics : FDA Approves Label Extension For Evrysdi For Infants With Spinal Muscular Atrophy

PTC Therapeutics Inc. (PTCT) said Tuesday that the U.S. Food and Drug Administration has approved a label extension for Evrysdi (risdiplam) to include infants under 2 months old with spinal muscular atrophy or SMA.

The approval was based on interim efficacy and safety data from the RAINBOWFISH study in newborns. The data showed that pre-symptomatic babies treated with Evrysdi achieved key milestones, such as sitting and standing, with half walking after 12 months of treatment. All infants were alive at 12 months without permanent ventilation.

Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal.

Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to distribute evenly to all parts of the body, including the central nervous system, and it is administered daily at home in liquid form by mouth or feeding tube.

Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group, and marketed in rest of world by Roche.

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