Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarep­ta Ther­a­peu­tics has filed the da­ta need­ed for an FDA ac­cel­er­at­ed ap­proval, which would be the biotech’s fourth if grant­ed by the agency.

The biotech has yet to com­plete con­fir­ma­to­ry tri­als for those first three con­di­tion­al nods. The fil­ing for its fourth Duchenne mus­cu­lar dy­s­tro­phy treat­ment, dis­closed Thurs­day, is not a sur­prise. Sarep­ta said in late-Ju­ly it would do so af­ter re­leas­ing pos­i­tive re­sults for the Roche-part­nered gene ther­a­py.

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Cambridge, MA, USA