Steady__T,
PDD Too small of a trial. Only one trial.
I don't see the submission of an NDA on a "tail end of doom indication" being stopped for trial size."
PD No trial at all for PD.
OK, but where is the trial? Are they reflecting and thinking on it? Trial design taking a year or 7?
AD Filing process started in EU.
Why not in AUS TGA and USA FDA too?
Adult Rett Small trial. I suspect the FDA would rather have a filing for pediatric and adult in one NDA.
I don't see the submission of an NDA on a "doom indication" being stopped for trial size. Nor for some scheduling preference. And pediatric patients would just get it after the adult approval, the issue is only insurance coverage. The logic of lets wait for two years or three for Pediatrics Retts to catch up before we submit on Adult Retts is stupidly cruel and economically wasteful. Why not in AUS TGA and EU EMA too?
Pediatric Rett Awaiting guidance from the FDA on the current trial. See adult Rett response above.
Submit the FDA NDA, EU EMA AMA and AUS TGA MAA with a letter from your Moms saying it works and a letter from the Moms who bailed on the placebo group during Covid. It should be enough that there is medically testable cellular changes in the treated group. Should at least be considered to receive Provisional Approval, perhaps EUA.
I say saturate the zone.
Bullish
News 
Market Data 
Discover 
