Some may recall on 2 March 2015 NEU announced that the FDA did not grant trofinetide Breakthrough Therapy designation for the treatment of Rett Syndrome- '
Breakthrough Therapy will not be granted at this time because the FDA considered that data from Neuren’s exploratory Phase 2 trial provide insufficient evidence to demonstrate substantial improvement over existing therapies using conventional statistical methods.'With the PMS P2 data more robust than Retts i would assume Breakthrough Therapy (BT)was applied for. NEU would not announce if they have or havent.
The below link contains some good information on what is BT. A few points to note:
* Historically, the FDA grants approximately one-third of breakthrough requests.This underscores the importance of a well-prepared application with compelling clinical evidence.
* Sponsors should submit a request no later than the end-of-Phase-II meetings
* The FDA commits to responding to breakthrough therapy designation requests within 60 days of receipt. Approval of the designation, denial, or a request for additional information.
I wonder if the additional information NEU is to provide to the FDA is also to assist in asssessing the BT application.
https://www.precisionformedicine.com/blog/what-is-an-fda-breakthrough-therapy-designation
(20min delay)