Genentech, a member of Roche, announced new long-term data for Evrysdi in a broad range of people aged 2-25 years with spinal muscular atrophy, or SMA, from the pivotal SUNFISH study. Data confirm increases in motor function were sustained at four years and the overall rate of adverse events continued to decrease over the 48-month period, reinforcing the long-term efficacy and safety of Evrysdi. Participants also reported continuous improvement or stabilization when independently performing activities of daily living such as picking up and moving objects. The data were presented at the Muscular Dystrophy Association, or MDA, Clinical and Scientific Conference, March 19-22. The increase in motor function from baseline observed during the first year of the study was maintained through the fourth year of treatment with Evrysdi, as measured by changes in Motor Function Measure 32 and Revised Upper Limb Module. Without treatment, natural history data show that patients with Type 2 or 3 SMA typically show a decline in motor function over time. Evrysdi was well tolerated over the four-year time period. Adverse events, or AEs, and serious adverse events, or SAEs, were reflective of the underlying disease. The most commonly reported AEs include headache, fever and upper respiratory tract infection. No treatment-related AEs led to withdrawal from the study. In addition to bringing Evrysdi to people around the world, Genentech also leads its clinical development as part of a collaboration with the SMA Foundation and PTC Therapeutics. Genentech is currently investigating Evrysdi in combination with an anti-myostatin molecule targeting muscle growth in the Phase II/III trial MANATEE for the treatment of SMA.
Published first on TheFly
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