Sarepta Sinks On Mixed DMD Gene Therapy Study
Blames 'Bad Luck In Randomization Process'
Any time advantage Sarepta's Duchenne muscular dystrophy gene therapy may have had over rivals, notably Pfizer, has disappeared after SRP-9001 failed to significantly improve muscle function in a closely watched trial.
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The company has initiated the first pivotal double-blind gene therapy trial in DMD in the US in a tight race with Pfizer.
Sarepta believes it is still very much in the running with its gene therapy but is playing down talk of an accelerated approval based on biomarker data.
Pfizer disclosed that a Phase III trial for the gene therapy has been unable to start in the US because of questions from the FDA, even while enrollment in 15 sites outside the US is under way.