St Jude Children's Research Hospital

FDA Expands Evrysdi Approval to Include Infants Under Two Months with Spinal Muscular Atrophy

- Roche and PTC Therapeutics' oral SMA drug Evrysdi (risdiplam) receives FDA approval for use in infants younger than two months, enabling treatment across all age groups from newborns to adults. - The RAINBOWFISH study demonstrated significant efficacy, with treated pre-symptomatic babies achieving key developmental milestones including sitting, standing, and walking within 12 months. - The expanded approval positions Evrysdi to compete directly with Biogen's Spinraza and Novartis' Zolgensma, while offering the advantage of at-home oral administration.