More dra­ma ahead: Sarep­ta plans to file for ac­cel­er­at­ed Duchenne ap­proval, with ad­comm a 'n­ear cer­tain­ty'

While Sarep­ta has three an­ti­sense oligonu­cleotide ther­a­pies ap­proved — al­beit not with­out con­tro­ver­sy — for Duchenne mus­cu­lar dy­s­tro­phy, it has been work­ing on a long-term treat­ment in the form of a gene ther­a­py.

Af­ter post­ing pos­i­tive re­sults for its Roche-part­nered gene ther­a­py, dubbed SRP-9001, ear­li­er this month, the Cam­bridge, MA-based biotech says it now in­tends to pur­sue an ac­cel­er­at­ed ap­proval for its lat­est Duchenne treat­ment. Sarep­ta shares $SRPT rose about 10% on the news in ear­ly Fri­day trad­ing.

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