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Gene Therapy: Four-Month Lag In Commercial Access Protects EMBARK Study, Sarepta Says

Executive Summary

Company’s prediction that Duchenne muscular dystrophy patients likely would not be able to access SRP-9001 until four months after accelerated approval eased some US FDA panelists’ concerns about interference with the ongoing confirmatory trial.

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Commercial Fate Of Sarepta’s DMD Gene Therapy Remains Tied To EMBARK

The company guided investors to expect a narrow initial label under a US FDA accelerated approval for SRP-9001, with expansion possible based on EMBARK.

DisEMBARK? FDA Questions Accelerated Approval Impact On Sarepta’s Fully Enrolled Clinical Trial

Advisory committee will consider how approval of SRP-9001 now might affect completion of the 52-week endpoint in EMBARK, a fully enrolled trial with two-thirds of the subjects coming from the US. Sarepta says all study subjects will have guaranteed access to the DMD gene therapy by end of September, making the risk of dropout ‘extremely low.’

The Ongoing Efforts To Confirm DMD Treatment Benefits

Infographic outlines the current status and history of several ongoing confirmatory trials for Duchenne muscular dystrophy treatments and a proposed gene therapy. Some have seen enrollment targets and eligibility requirements change over the years, along with the estimated completion dates.


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