FDA Pushback on Accelerated Gene Therapy Approvals

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:

1. Lagging confirmatory trials
   
   
2. Outdated safety monitoring plans
   
   
3. Poor communication across teams and with FDA
   
   

She offers practical guidance to avoid regulatory setbacks:

• Build flexible governance frameworks
  
  
• Stay proactive with FDA engagement
  
  
• Plan for legal and regulatory risks in confirmatory trials
  
  
• Prepare for public, regulatory, and payer scrutiny post-approval

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.

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