FDA Pushback on Accelerated Gene Therapy Approvals

DarshanTalks Podcast

Content provided by Darshan Kulkarni. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by Darshan Kulkarni or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://podcastplayer.com/legal.

1M ago 4:10

MP3•Episode home

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Outdated safety monitoring plans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 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.

Like, comment, or reach out to learn more.

Support the show

265 episodes

#Science #Society #Biology #Documentaries #Podcasting Education #DarshanTalks #Pharma #Medical Device #FDA #Law #Regulations #Guidance #Clinical Research #Advertising #Mergers #Acquisitions #M #Pharmacy