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.
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,...
In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.
Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.
Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.
