Sarepta Therapeutics reported that its long‑awaited ESSENCE confirmatory trial of two exon‑skipping drugs missed the primary endpoint, producing results that the company described as influenced by pandemic‑era missed doses. The company plans to press the FDA for full approval despite the negative outcome, while investors reacted sharply to the readout. The development raises questions about the durability of accelerated approvals contingent on confirmatory evidence and illustrates regulatory and commercial risks for antisense and exon‑skipping strategies in rare neuromuscular disease.