European Commission approves Roche ’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA)

Evrysdi available to treat people of all ages with SMA in the European Union, including babies from birth1Approval is based on interim data from ongoing RAINBOWFISH trial showing majority ofEvrysdi-treated babies were able to stand and walk within timeframes typical of healthy babies by 12 months ’ treatment2,3Evrysdi is the only non-invasive SMA therapy and is approved in 100 countries with more than 11,000 patients treated globallyBasel, 29 August 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission has approved the extension of the Evrysdi ® (risdiplam) European Union (EU) marketing authorisation to include infants with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months.1 Interim data from the ongoing RAINBOWFISH trial in pre-symptomatic babies from birth to six weeks with Type 1 SMA supported the marketing authorisation extension.“The SMA community welcomes the European Commission’s decision to extend the use of Evrysdi from birth,” said Dr Nicole Gusset, President& CEO of SMA Europe. “Preserving motor neurons from the earliest age possible and preventing their irreversible loss can have a substantial impact on a person’s future ability to move and function. We look forward to continued collaborative efforts to improve diagnosis, including newborn screening, and ensuring all individuals living with SMA have access to medicines.”“With this label extension...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news