Nippon Shinyaku Co., Ltd.’s failed confirmatory trial for the Duchenne muscular dystrophy treatment Viltepso (viltolarsen) is sure to reignite questions about the use of dystrophin as a basis for accelerated approval, as well as the ability of selected functional endpoints to detect change in the disease.
On 27 May, Nippon Shinyaku announced that RACER53, a Phase III, placebo-controlled study of 77 boys with ambulatory DMD, failed its
Key Takeaways
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The viltolarsen confirmatory trial failed its primary endpoint of time to stand from supine at 48 weeks.
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RACER53 is the second confirmatory trial of a DMD accelerated approval treatment to fail its primary endpoint, and the first of three confirmatory trials for four marketed antisense oligonucleotides to report top-line results
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