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Roche gets USFDA nod for spinal muscular atrophy therapy Evrysdi for babies below 2 months
Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently.
Basel: Roche has recently announced that the U.S. Food and Drug Administration (USFDA) has approved a label extension for Evrysdi (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA).
The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. Evrysdi is now approved in the US to treat SMA in children and adults of all ages.
Read also: Roche rolls out 3 unique LightMix Modular Virus kits for monkeypox
Ruchika Sharma joined Medical Dialogue as an Desk Editor for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751