Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA)

Basel, 28 September 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi ™ (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support for at least 5 seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III). Sixty-five percent (11/17 vs. 9/17 at 1-year) had maintained upright head control, 29% (5/17 vs. 2/17 at 1-year) could turn themselves over and 30% (5/17 vs. 1/17 at 1-year) were able to stand either supporting weight or with support. After two years of treatment with E vrysdi, 71% (12/17 vs. 10/17 at 1-year) of infants achieved a CHOP-INTEND* score of 40 points or more and all infants increased their score from month 12 to month 24. Of the infants alive at two years (n=14), 100% maintained the ability to swallow and 93% (13/14) were able to feed orally. Safety for Evrysdi in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified. The most common adverse ev...
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news