Bought 10k shares this AM. At this point is there still a chance that FDA will allow them to file an NDA next year or is it certain that a full P3 is required? When would we find out how the FDA is going to go? It seems there is a lot of pressure from parents to get this approved as there is no signif toxicity and at least reasonably strong evidence of efficacy. Does the FDA not accept the presence of truncated dystrophin as proof of efficacy?
How well did the kids in the P2 trial of the Glaxo drug do? Was it comparable to SRPT data? Does anyone have a link for that data so I can check it out directly? Thanks for any help.
I know this is a serious inquiry, but it reminds me of a scene from dumb & dumber.
Loyd: What do you think the chances are of a guy like you and a girl like me... ending up together?
Mary: Well, Lloyd, that's difficult to say. I mean, we don't really...
Lloyd: Hit me with it! Just give it to me straight! I came a long way just to see you, Mary. The least you can do is level with me. What are my chances?
Mary: Not good.
Lloyd: You mean, not good like one out of a hundred?
Mary: I'd say more like one out of a million.
Lloyd: So you're telling me there's a chance... *YEAH!*
I think there's a chance, but that chance depends on continuing stability being demonstrated on the open label extension. And I have a hunch that GSK will find sub populations that look good for D. That would dispel the notion that exon skipping doesn't work. Either one of these increase the odds substantially.
I have read the entire Mendell article from Annals of Neurology and the Cowen report on SRPT. I am a physician, but I am not a pediatrician or a DMD specialist and the FDA is not something I have an expertise in other than the routine knowledge that doctors in general have. I think SRPT is a good purchase here as I am willing to wait 3 years if that's what it takes to get a P3, but I wanted to sound out any other views that may be out there. Anonymous internet boards are a very poor source of useful info but there are often a handful of well informed posters with good analysis to offer. Just looking to see if there might be some here. Whether you believe that I bought 10k shares or 10 doesn't matter, to me or anyone else.