FDA Gives Green Signal To Pfizer's Duchenne Gene Therapy Trial

After the FDA lifted its hold on a late-stage study, Pfizer Inc PFE said it would open the first U.S. trial sites for its experimental gene therapy for a muscle-wasting disorder.
The FDA had put Pfizer's Investigational New Drug (IND) application for fordadistrogene movaparvovec on hold, and Pfizer has addressed the agency's requests related to the potency assay.
The global Phase 3 study, CIFFREO was paused in December 2021 to implement a protocol amendment following a fatal serious adverse event in a Phase 1b study in the non-ambulatory cohort.
Related: Pfizer's Duchenne Gene Therapy Trial Put On FDA Hold After Patient Death.
Pfizer said the regulatory clearance came after data reviews and tweaks to the trial to include a seven-day hospitalization period to monitor patients receiving the gene therapy closely.
Big Pharma said it would have nearly all late-stage study sites open by the end of June.
Price Action: PFE shares are up 1.05% at $50.26 during the market session on the last check Thursday.