FDA Pushback on Accelerated Gene Therapy Approvals

Send us Fan Mail In this episode, Edye Edens dives into the FDA’s evolving scrutiny of accelerated approvals in gene therapy and rare diseases. Using the example of Sarepta’s muscular dystrophy treatment, she explains how postmarket safety issues and incomplete confirmatory trials can put previously celebrated approvals under renewed FDA evaluation. Edye breaks down three common pitfalls sponsors face: Lagging confirmatory trials Outdated safety monitoring plans Poor communication across te...