Pfizer DMD Gene Therapy Phase III Trial Stalled In US After FDA Questions On Potency Assays

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.

FDA stop sign. Sarah Silbiger/Getty Images — • Source: Sarah Silbiger/Getty Images

Pfizer Inc.'s gene therapy for Duchenne muscular dystrophy (DMD) now faces a delay with the start of a Phase III trial in the US due to outstanding questions the FDA has related to "technical aspects" of the "potency assay metrics."

Pfizer has been the first to advance its gene therapy candidate – fordadistrogene movaparvovec – into Phase III clinical development,...