Chasing Pfizer, Sarepta Prepares To Launch Pivotal Duchenne Gene Therapy Trial
Investors Still Skeptical
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.
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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.
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.
Bepirovirsen made the hepatitis B virus undetectable in nearly 30% of patients in a Phase II study – but it remains to be seen whether this response be sustained once treatment with the novel antisense product ends.