Four-year follow up data for Roche ’s Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk

Data from ongoing FIREFISH study confirm long-term efficacy and safety profile ofEvrysdi in children with Type 1 SMANinety-one percent of children were alive at month 48More than 95% maintained the ability to swallow- without treatment they would have required feeding support and majority would have died within 2 yearsEvrysdi is now approved in 99 countries with more than 8,500 patients treated globallyBasel, 30 June 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new long-term data for Evrysdi ® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing its sustained efficacy and safety profile in children with Type 1 spinal muscular atrophy (SMA). FIREFISH is a two-part study in babies aged 1-7 months at the time of enrolment. After four years of treatme nt with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support. All the Evrysdi-treated children who were alive at the time of the primary analysis were still alive at month 48.Additionally, the majority of infants maintained their ability to feed by mouth and swallow up to month 48. Motor function was assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2) and abilities were either maintained or improved over four years of Evrysdi treatment. Without treatment, children with Type 1 SMA are not expec...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news