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.
Accelerated Approval: Nippon Shinyaku’s Viltepso Fails DMD Confirmatory Trial, Raising Questions
The Duchenne muscular dystrophy treatment missed its primary endpoint of time to stand from supine in the RACER53 study, raising more questions about using dystrophin changes to support accelerated approval and the ability of functional endpoints to detect clinical change.
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