Sarepta Crashes After Two Drugs Fail Confirmatory Tests

Sarepta Crashes After Two Drugs Fail Confirmatory Tests

Sarepta stock crashed late Monday after the biotech company said two of its muscular dystrophy drugs failed their confirmatory studies.

Sarepta’s Amondys 45 and Vyondys 53 confirmatory trial fails to 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 fu…

Sarepta's Duchenne confirmatory trial fails, but biotech will ask FDA for full approval anyways

Sarepta Therapeutics’ long-awaited confirmatory study of two of its ‘exon-skipping’ therapies for forms of Duchenne muscular dystrophy failed to meet its primary endpoint, the company announced Monday. But despite that, the company plans to ask ...