Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval

Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval

Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available under accelerated approvals. The post Despite Phase 3 Failure, Sarepta Still …

Sarepta Duchenne drugs come up short in confirmatory test

Still, CEO Doug Ingram claimed removing the two drugs, Vyondys 53 and Amondys 45, from market would make “little sense” and that the evidence accrued to date makes a strong case for full approval.

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.

Two of Sarepta’s DMD drugs fail to meet primary endpoint in confirmatory trial

Despite the trial failure, Sarepta will still seek full approval of AMONDYS 45 and VYONDYS 53 in DMD from the FDA.The post Two of Sarepta’s DMD drugs fail to meet primary endpoint in confirmatory trial appeared first on Clinical Trials Arena.

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 ...