A long‑ awaited confirmatory trial for Sarepta’s exon‑skipping therapies in Duchenne muscular dystrophy failed to meet its primary endpoint, the company announced, dealing a significant regulatory and commercial blow. Sarepta said it will nonetheless pursue full approval discussions with the FDA based on the totality of evidence, including real‑world and historical data. The readout and the company’s response were covered in company statements and biotech press reports. The confirmatory miss raises questions about accelerated approval pathways tied to surrogate markers and the evidentiary burden for post‑approval confirmatory trials. Sarepta’s strategy to seek full approval despite negative confirmatory results will test FDA precedent on how to weigh clinical outcomes versus accumulated real‑world evidence.