Sarepta Therapeutics reported that a long‑awaited confirmatory trial of two exon‑skipping therapies for Duchenne muscular dystrophy did not meet its primary endpoint. Company leadership said it will nonetheless press the FDA for full approval, arguing that the totality of evidence supports the therapies’ benefit, even as investors and analysts questioned the data and management’s handling of the program. The failure reverberates across the rare‑disease and gene‑therapy sectors: confirmatory studies remain pivotal for accelerated approvals, and negative readouts can trigger regulatory re‑assessment of conditional authorizations. Sponsors developing transformative but uncertain therapies may reassess post‑approval evidence plans, and regulators will continue scrutinizing the robustness of surrogate endpoints and external control comparisons.