A long‑awaited confirmatory trial for Sarepta’s exon‑skipping therapies failed to meet its primary endpoint. The readout affected Vyondys 53 and Amondys 45 and sent Sarepta shares tumbling; management says pandemic‑era missed doses and trial disruptions affected results and will press the FDA for full approvals based on the totality of evidence. The outcome reignited criticism about accelerated approvals based on surrogate or biomarker data and renewed calls from some stakeholders for stricter confirmatory standards.