If you replace a missing element in a disease that causes that disease the drug clearly has a major benefit .I would also say that with a outstanding safety profile what more could the FDA want..
All the other companies ,we won't mention any names'' cannot dose high enough to get a stable effect because of their drug platform due to toxic limits.
Could be once all the moves are made from many pockets the FDA will let this go without the delay you think..Remember everything is still on the same time frame..
Would it be a difference between a ultra orphan approval drug vs a plain orphan drug?
seems like in the usa it is just that.....
GILD WORTH AROUND 120.00 A SHARE BY END 2014
also could not find a better solid investment by my opinion .Other stocks to jump on for 2014 ride up
atrs...cers..and srpt more than likely...........
Sarepta's is the one long term dosing.......................flat out..
FDA has reached no conclusions about the possibility of using accelerated approval for any new drug for the treatment of Duchenne muscular dystrophy, and for eteplirsen in particular. [Emphasis added.]
i believe they just rolled some into 2014 for approval or to kinda spread it out. They really seem not to sure of themselves it appears. They also are showing some restraint on purpose ''preventing over done responses''
You can always say that you have doubts but these kids that are on Sarepta's drug and the parents of these kids show no doubts ,,so who do you believe? very simple investment common sense.
It is plainly stated that drugs for rare diseases could be approved based on a surrogate endpoints or any ''other facts'' the key here is ''other ''