MedCity News June 13, 2024
A Pfizer gene therapy for Duchenne muscular dystrophy failed its Phase 3 clinical trial. But analysts draw distinctions between that therapy and Sarepta Therapeutics’ Elevidys, which awaits an FDA decision that could grant it full regulatory approval.
A Pfizer gene therapy for the rare muscle-wasting disease Duchenne muscular dystrophy did not help patients’ ability to walk or stand up in a pivotal clinical trial. The pharmaceutical giant is still weighing its next steps for the therapy, but analysts say these disappointing trial results, the latest in a series of setbacks, likely mark the end for this program.
The Phase 3 test enrolled boys ages 4 to 7 who still had the ability to walk. The trial’s main goal was to...
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