Sarepta’s Amondys 45 and Vyondys 53 confirmatory trial did not achieve statistical significance on primary endpoint

Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53.  The study did reinforce the favorable safety profile of the therapies, but did not achieve statistical significance on the primary endpoint  (4-step ascend velocity at 96 weeks). 

What comes next?  Sarepta says it plans to discuss with the FDA a path from accelerated approval towards full approval, utilizing this data as well as real world evidence of these therapeutic approaches collected over multiple years.  

Link to press release: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-third-quarter-2025-financial

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