RettEd: What Comes Next? The FDA Regulatory Process from NDA to FDA Decision and Beyond

Original Air Date: Monday, August 1, 2022
With the recent news about trofinetide’s new drug application (NDA) submitted to the FDA, we are entering a new space for Rett syndrome: FDA Review. While this is the first time for Rett, it won’t be the last, and our community has a lot of great questions:
“What does submitting an NDA mean? What’s the next step? How much longer? When will we have an approved treatment? How will my loved one get access to this treatment?”
To answer these questions and many more, IRSF is excited to bring back FDA expert, James Valentine, Esq., to join IRSF’s Chief Science Officer, Dr. Dominique Pichard for a live webinar. Join them as they review the FDA regulatory process, share what happens between a successful Phase 3 clinical trial and an FDA decision about a drug or therapeutic, and explain what could come next for trofinetide and the Rett community.
Thank you to our RettEd Empowerment Sponsor, Acadia Pharmaceuticals, for helping IRSF provide educational resources and support to families affected by Rett syndrome.
Medical Disclaimer: All information, content, and material provided in this webcast is for informational purposes only and is not intended to serve as a substitute for the consultation, diagnosis, and/or medical treatment of a qualified physician or healthcare provider. Always seek the advice of your physician or other qualified healthcare provider with any questions you may have regarding a medical condition.
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[@hishamhittini5069]

Can we get update on the process outcome

[@ericaobrien5941]

I feel like this could have been a two page FAQ. 🥴 This video was marginally helpful, but too long. Most folks are visual learners. It was difficult to sit through the extended dialogue....and I'm a doctor. I've sat through plenty of hours worth of courses in my lifetime.
The FDA sounds like the epitome of a bogged down bureaucracy. I'm sick and tired of hearing about all of these reasons why we are still waiting on a potentially life-changing pharmaceutical for our Rett kids (and Rett adults!).
Sincerely,
A very tired, frazzled, and out-of-patience mother to a Rett girl.

[@3orangewhips71]

Thorough regulatory compliance and evaluation is a must in this process. We don't need another Cutter Incident or similar.