From FDA's website: 'Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session. The contact person will notify interested persons regarding their request to speak by April 4, 2016.'
This would be most unfortunate, but likely, and could limit the number of expert DMD witnesses.
it is generally difficult to convincingly demonstrate levels of dystrophin below 10% of normal levels."
Since this is the case, and it's quite possible that levels of D could be in the 3-5% range, which would be clinically significant, how then can the FDA stand confident that 0.9% is a definitive number? It can't be.
Not sure why they would be any different than BioMarin's briefing doc questions. And if they aren't, it seems like its another drive by mugging by the FDA. I think they are trying to kill off exon-skipping as a class.
Then why was there chatter by the FDA & Srpt that the small N wasn't an insurmountable obstacle?
FDA encouraged the sponsor at the March 2013 meeting to conduct an adequately powered placebo-controlled trial of eteplirsen, stating “if it is true that eteplirsen leads to remarkable clinical benefit in even some patients, there is no doubt that a feasible placebo controlled study can be designed to demonstrate that benefit.” FDA also stated that “there is considerable variation among individual patients with regard to clinical measures and important milestones” and that data from an open-label study “may only be interpretable if a relevant objective endpoint obviously insulated from bias demonstrated compelling data that are clearly outside the know variability range for DMD.” FDA further stated that, at that time, comparison of data from Study 202 did not provide interpretable evidence of benefit “given the limitations of the open-label design for protecting against bias on effort-dependent endpoints like 6MWT.” At a July 2013 meeting with the applicant, at which the possibility of NDA filing based on dystrophin production was discussed, FDA similarly expressed reservations about natural history controls “due to the usual difficulty in showing comparability between the study populations in natural history studies,” and reiterated that 6MWT was susceptible to bias in the proposed natural history comparison.
Despite of this rebuttal, and after reviewing many other significant questions/comments within the briefing docs, it looks like an extreme long shot for AA, even given the safety profile. Maybe the best we can hope for are positive read outs on the confirmatory trials, because it seems that the FDA wants to see a larger data set.
Will help support the sp for a while, assuming whatever % of shorts cover.
The briefing docs suggest that FDA won't AA Etep, but the path forward will be: "Let's revisit this when you have more data."
One of the disturbing things in the briefing docs stated that Srpt digitally altered some of the D slides to make it appear that D production was more robust than it actually was. At that point, I bailed on all of my shares. Really hated to do that, but I'll wait until the dust settles before making a decision to get back in, or not.
Speed, you've it it out of the park....again. Great post and excellent summary. And, the most "thumbs up" I've seen in many moons - well deserved!
True, but a big splash on BB helps the masses to digest a fundamental truth about the likelihood of approval. I'll take it.
What the basis of your belief? Just a hunch?
It could be that FDA has the opportunity to show their "flexibility" in drug approval and wants to showcase Srpt AdCom & AA as a manifestation of this process. The panel might not be 100% convinced about efficacy with the small N issue looming front and center, but they will be able to suspend scientific doubt with all of the other supporting evidence AND safety.
In the end, the FDA will walk hand-in-hand with Srpt to the finish line. Nice PR move, but it's also the right thing to do, after all the waffling over the past 2 years by FDA, while they were getting up to speed on DMD.
Let's keep that in mind, which will exacerbate volatility.
Also keep in mind that BMRN was $95 in pre-market yesterday, and then the big leak happened.
Elliot Favus came out this morning with another short attack; no surprise there, so that's causing some ripple.
Simp is predicting "ugly briefing docs" next week.....boo!
Expect typical scientific scrutiny; nothing "ugly" about that.
Parent testimony alone will not overcome bad data. BMRN
Parent testimony alongside meaningful data will be supportive of positive AdCom at end of day.
The totality of data on Etep + pristine safety will lead to AA/Conditional Approval.
This gets the ball rolling toward full approval when confirmatory trial's results are in.
Expect more of the same: safety/ stabilization/ D production/ pulmonary benefit.
It would be irresponsible for FDA to not grant AA for Etep.
Worry not about share price noise between now and AdCom.
Bionerd 51 and a host of other longs with scientific background have been right at every turn. Big HT to them.
Sentiment: Strong Buy
Gotta remember that Srpt has a small place in the IBB and when these kinds of ETF's get slammed it affects all of the stocks within the index, and folks who have sell stops often get triggered, which leads to oversized moves to the downside, furthering more selling by nervous nellie day traders. The solid retail holders here aren't budging, and some are adding on these kinds of sell-offs.
Easy. The street always saw these two drugs as "similar", so in that mind-set what's bad for Bmrn is bad for Srpt. The bear trap is getting set to put the hurt on the most shorts.
I think the secondary will be to support ADP trials, which will take a lotta $$. And, that much cash on hand will make ACAD an even juicer takeout target, no?