Sarepta’s confirmatory Duchenne trial fails to meet endpoint, complicating future for controversial drug

Sarep­ta and Roche’s con­fir­ma­to­ry study for a Duchenne gene ther­a­py failed to hit its pri­ma­ry end­point, com­pli­cat­ing plans to con­vert the drug’s la­bel in­to a full ap­proval for a wider pop­u­la­tion of boys.

In the Phase III EM­BARK study, 125 pa­tients re­ceived ei­ther place­bo or Ele­v­idys, the gene ther­a­py de­vel­oped by Sarep­ta. Af­ter one year, those who re­ceived Ele­v­idys saw a 2.6-point im­prove­ment com­pared to a 1.9-point im­prove­ment in the place­bo group on a func­tion­al mo­bil­i­ty scale called the North Star Am­bu­la­to­ry As­sess­ment, the com­pa­nies said Mon­day.

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