But it's not the ESSENCE trial you mean because ESS isn't exon51. And the large newish exon51 trial (whatever it's called!) isn't a placebo trial - so the whole lovely argument (mine and yours) is moot. (I can't be expected to keep track of all these blasted trials we've got going!)
Possibly, but that's not very logical. Because with an approval decision likely parents are already on hold and will stay that way until a decision. So that boat has sailed. (Unless the FDA has already decided they will not approve and just are stalling because they are gutless).
28 weeks plus 212 weeks is 240 weeks. Feb 2012 thru Feb 2016 is 208 weeks. It's now been about 28 weeks since then, so yes, 236 weeks. So in 4 weeks the trial and dosing ends. Do you think the FDA is actualy going to drag their feet and push it right to the limit? I would imagine they'd give us a month to get the drug packaged and ready to arrive where it needs to be (at least to the original 12 boys) in time for their next weekly dose after the trial ends October 6th or thereabouts. I'm expecting a decision over this week-end or next week.
Actually, he's surprisingly good! Which just goes to show they are willing to pay top dollar to discourage new (old oldish, maybe) investors in this company. Wonder why? It's all a drop in the bucket but even then it's worth it to them to have him here doing what (little, probably) he does ....and pretty well too, in my opinion, actually. We should be flattered we rated one of the best in the biz.
Eleven years later and after hundreds of published studies from probably a hundred or more researchers coming at DMD via PMOs from scores of different angles, and after dosing the drug for over 5 years in the original 12 boys and numerous since, I think the FDA can feel confident that this is a real drug - a real science. In fact at this point the science has matured to the point that it's essential done. It's here, FDA, whether you like to admit it or not. It's for real. Release the drug!
Hopefully, they've been getting their hands on a few tech tweaks others have discovered or disease targets. I suppose there's no need to spell them out at this point.
I'm with you on the Labor Day deadline. That prediction may not play out but this one will: when they do approve there will be no cheering, no smiles, no happiness. The interminable delays have buried any of that kind of thing. Disgust rules the day now and it's not going to go away for a long long time.
Good letter but I wish she hadn't gratuitously used the word "systemic". "Going forward" this willy-nilly use of "systemic" is just not "sustainable".
Change at Neurolgy is all we really need. And everyone knows what kind of change is needed. Personnel. Systemic change? Not sure what that would mean. We need personnel change. The system is probably fine - it's the people who ignore it and try to gum it up that are the problem. Simply substituting honorable dedicated efficient PEOPLE (not folks) the Neology Department could be adequately reformed.
Maybe Woodcock and Califf are actually just the usual wimps - letting anybody and everybody push them around. Scared. Califf looked good in his previous (academic leadership) position, but maybe he can't hack it in this setting. Could be. This is probably the best test we'll see of that. If he fails now he might as well hang it up.... and let Trump find a leader with some real courage and determination to shake up that mess over there.
Woodcock and Califf need to show they can lead. Otherwise they'll be disrespected marshmallows in the eyes of all FDA employees. They need to knock some heads here and stop #$%$-footing around. It's getting embarrassing now.
She doesn't expect to pick up one vote by anything she says. She just has to say things when they tell to it's time to say things. She'll get the same amount of votes if she reads from Numbers in the Bible for the next 80 days. Everybody knows that.
No no. I always put fuzz around my August 23ish dates. Ultimately Labor Day - a few days before or aft is my limit. Anything after that I give up.
Lots of naps too. And those splitting headaches. Oy veh! In her mind I think she's imagining that her Presidency ends after election day. From there on in it'll just be 4 years of meals, naps, and doctor visits. Or maybe she'll just retire early. I mean once you've achieved "first women president" what's left to do? Not much really. In HER mind. Let's get real. You know that's how she's thinking about this whole stupid thing.
Saturn: "This is a DPO situation...
This could end in 5 minutes or two months from now"
Actually you're off a bit on that. The reason is in the quote from the fda rules that I posted yesterday. All players are supposed to take into consideration such "deadlines" - so 90 days from the May 24 pdufa day delay probably is considered to be one such "deadline" that they are supposed to wrap a DPO situation by. We will see if they can do it today, but I think they'll try and do it as close to today as they can.
Because there is no way to market a placebo. Eteplirsen is the next best thing.
Jeff, went and read it all again. It's basically just as we've been saying - 35 days plus or minus. The if Farkas appeals to the science board more time.....but how much time?..... here's a quote from that process:
"Note regarding timing: because resolution cannot be predicted at any given
management level, it is important to move as quickly as possible throughout the
process, regardless of whether an informal or formal process is invoked. This is
particularly important for a scientific dispute that involves a pending product approval
or clearance action. To the extent possible, staff should be mindful of any relevant
regulatory review time frames"
N.B. that there can also be either a "formal" or an "informal" process here.
Again I'll say that that is where we are now - or that part has just concluded and we'll get our answer soon.
Jeff - so what is the shortest it could take. (The Woodcock side of things doesn't have to take all the time they are allowed).