EU Committee for Medicinal Products for Human Use (CHMP) recommends Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA)
July 28th, 2023
The EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi® (risdiplam) European Union marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, including from birth to below two months. The recommendation is based on an interim analysis from the ongoing RAINBOWFISH trial in pre-symptomatic babies with Type 1 SMA from birth to six weeks. In SMA, early treatment is critical to counteract ongoing and irreversible loss of motor neurons. A final decision regarding the approval is expected from the European Commission later this year. For more information, please visit www.roche.com.
Categories: Research
