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:
She offers practical guidance to avoid regulatory setbacks:
Accelerated approval is a major milestone—but it’s not the end. It's the beginning of the next regulatory chapter. Tune in to learn how to manage post-approval responsibilities and secure long-term success for your therapy.
Like, comment, or reach out to learn more.
