CHMP recommends Roche ’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA)

Positive recommendation is based on interim data from ongoing RAINBOWFISH trial which showed majority ofEvrysdi-treated babies were able to stand and walk within timeframes typical of healthy babies by 12 months ’ treatment1,2If approved by the European Commission,Evrysdi will be available to treat people of all ages with SMA in the European Union, including babies from birthEvrysdi is now approved in 100 countries with more than 8,500 patients treated globallyBasel, 21 July 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi ® (risdiplam) European Union (EU) marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to fourSMN2 copies, including from birth to below two months.1 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.3,4,5 A final decision regarding the approval is expected from the European Commission later this year.“Treating babies with SMA early helps them to carry out daily activities such as sitting, standing, and walking,” said Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product Development. “This CHMP recommendation is ...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news