Top FDA official overrules staff to approve gene therapy that failed trial

The FDA approved the gene therapy Elevidys for Duchenne muscular dystrophy, despite it failing a Phase III trial and facing objections from FDA staff. The decision was primarily made by Dr. Peter Marks, overruling expert review teams and directors. Elevidys was initially approved last year for specific age groups under an accelerated process but has now been granted traditional approval for all DMD patients. Critics, including pharmaceutical experts, have raised concerns about the approval process, suggesting a shift towards emotional rhetoric over scientific evidence. Internal documents reveal a clear divide between Marks and FDA staff, with disagreements on the therapy's effectiveness based on trial results. While FDA statisticians and other teams expressed doubts, Marks found the secondary endpoints convincing enough to support the therapy's approval. The decision to approve Elevidys has sparked criticism and raised questions about the FDA's approval standards and the influence of high-ranking officials on regulatory decisions.