You're an idiot. Sarepta did what was considered more than reasonable testing with Dr. Mendell as the lead doc. His quanatifications were questioned by the FDA, and when the slides were re-read at three independent laboratories, those results confirmed Dr. Mendell's original findings, thus strongly supporting his conclusions, as well as methodology. In FACT, the net change noted by the three different laboratories was actually greater than that noted by DR. Mendell.
The FDA is, and was, the true problem in the DMD arena. They chose to form conclusions about Eteplirsen's effectiveness BEFORE actually have the data in hand. They threw Dr. Mendell's findings under the bus, and then looked foolish when he was vindicated. They REFUSED to provide absolutely critical guidance on P3 trial design, or when they did provide guidance, it was for an unethical, impossible to enroll placebo controlled P3, which SRPT rightfully declined to initiate knowing that A) it would be highly unethical and B) likely impossible to enroll even if they tried. This contention is now supported by the difficulty the company is having in fully enrolling their current studies.
The petitions were not promoted by SRPT, nor were they initiated by SRPT. Blaming Sarepta for actions taken by others is ridiculous, and inaccurate. Go ahead and prove any Sarepta involvement in publicly lobbying the FDA. You won't because you can't. Just like the other shorts, you make #$%$ up in order to fit your ugly agenda. FActs be damned. BTW, Immunoflourescence is NOT #@*& testing, as you suggest. In fact, DMD experts all agree that it is equally important, if not more so, than Western Blot. Nice try. And Sarepta could NOT provide a "much better" testing protocol as you incorrectly suggest, because the FDA refused to provide guidance for TWO YEARS!
Next time, perhaps you could do better research before polluting the board with your uninformed, erroneous guesses. I won't hold my breath though.
4% might be as easy as putting some hexose into the infusion solution, but we'll never know unless the FDA stops playing games and grants approval. Or, the kids might have to take dantrolene on their infusion days, and voila!, 4% or more. We won't know without an approval, which will open up doors to methods that improve the impact that PMO's can have.
Uh......it's ten patients according to your logic. Better yet, check out the chart between the four in the placebo arm who crossed over and the six you reference who were on the drug from the beginning. THIS is one of the strongest efficacy signals that Eteplirsen has. One can CLEARLY see (not you cuz you REFUSE of course) that the placebo kids were deteriorating rapidly, as would be expected. And then? VOILA! They stabilize and begin behaving in a very similar manner as those boys fortunate enough to be on the drug from the beginning.
POSITIVE DRUG IMPACT - PROVEN
What else is needed to warrant approval? Allow me to help you, since we both know you need it and it's a rhetorical question: NOTHING more is needed given the flexibility granted in FDASIA.
Now run along and try to deceive folks somewhere else.
He better go ahead and correct the "mistakes" that he "accidentally" included in his BD so that he could significantly diminish the positive drug effect that shows up in the un tainted data. Of course, then his anti Eteplirsen argument is that the data came in a way he hates even though it shows the drug works. He'll get crushed.
John Fierce is a complete TOOL. Einstein could come back from the dead and prove to him that Eteplirsen works, and Fierce would say Einstein wasn't smart enough to know for sure. Anyone who listens to that guy is out of their mind.
"as timely a manner as possible"...........after already wasting an extra three months with the rescheduled Adocm, does seem to imply that it won't take very long. And given the almost unconscionable amount of time that the FDA/DNP have previously wasted, taking an additional two months would be almost unforgivable. If nothing else, these boys and their families DESERVE an answer to the question. "YES".....or "NO". It shouldn't be this hard.
The Adcom panel was shown in multiple instances, information or data that was completely false. They were shown controls polluted with higher functioning exon deletions/Beckers too. They would be basing their opinion, as they were previously, upon seriously flawed data unless JW also first corrected the deception that the DNP used to make their ultra biased presentations.
"I'd like to ask you, IF the Department of Neurology presentation had not included numerous blatantly incorrect or false statements, would you have voted differently?" JW already knows the answer to that question and I bet she has WAY better things to do than go back and ask Adcom members rhetorical questions.
I place little value in that stat. How many of those went through the DNP and were then approved. All FDA divisions are not equal. The only one I know of is BMRN/Drisa and they got a CRL.
Reading through all stop codons instead of just the ones associated with a particular disease is the antithesis of precise. In fact, it's not a good idea, because all the ones not associated with the target disease are not in need of a read through. They are there for a reason. So..................of to ignore for you.
Yeah, in ten years, MAYBE. And then they get to try to drive their NDA through Hell.....I mean the DNP. Man, you're a dope neo. Always have been. Get lost please.
Ya, funny how they fooled ALL of the DMD experts in the world to , huh?
"The FDA has communicated that they will continue to work past the PDUFA goal date and strive to complete their work in as timely a manner as possible."
That , to me, means days not weeks.
Well, the shorts have been getting information before the rest of us for quite some time, and my guess is it's oozing out of the DNP. Too bad the SEC doesn't give a darn.
My guess...........................18 days or less. The FDA stated that they intended to get the decision out as quickly as they could. The BMRN delay was 18 days, so they are capable of getting it done in that time frame of less.