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

• After the FDA lifted its hold on a late-stage study, Pfizer Inc (NYSE: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.