PTC Therapeutics says CHMP adopts positive opinion for Evrysdi

PTC Therapeutics announced that the Committee for Medicinal Products for Human Use has adopted a positive opinion for the extension of the Evrysdi marketing authorization to include infants under two months of age in the European Union. A final decision regarding the approval is expected from the European Commission later this year. The CHMP opinion is based on the RAINBOWFISH interim analysis, which included six babies with two or three copies of the SMN2 gene who completed at least one year of study assessments. Of these, 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% could walk independently. All infants were alive at 12 months without permanent ventilation.