After a meeting yesterday with the FDA, Sarepta Therapeutics plans to submit a rolling New Drug Application (NDA) for eteplirsen beginning next week and wrapping up the application by the middle of this year. This is great news, and it would not have happened without all your work and support, so THANK YOU.
We have been waiting far too long for this step, and there are many more steps that will follow. Once the complete NDA is submitted, the FDA has 30 days to decide whether it will “file” it or officially accept it. Then it will take the FDA up to six months to review it, including possibly convening an independent Advisory Committee.
The FDA has been reviewing data on this drug for over two years now, so it’s time to move quickly instead of using the full time periods allotted for each step. We look forward to a transparent and expeditious process going forward. Remember, eteplirsen only treats 13% of the Duchenne population, so there is much work remaining to develop similar therapies for all kids who can benefit. We expect the FDA and the company to collaborate expeditiously to get these follow-on drugs developed as quickly as possible.
We are closely monitoring this process and will make sure to keep you informed as it progresses.
Again, THANK YOU for your help and commitment to our boys!
Sincerely,
THE RACE TO YES
@TheRaceToYes