. . . if it has clearly delivered on dystrophin, it's AA . . . if it hasn't or the new results are ambiguous, then it's a CRL with another dystrophin review sometime down the road . . . which is more likely? . . . still a coin toss, imo . . . (still holdin' my shares and hopin' for $60 and a quick exit) . . .
. . . well, rl, I suspect they are working on a CRL -- so as to provide the company with a clear direction on a path forward for etep . . . it's a lot of work because it covers a lot of ground, and it has to be comprehensive . . . but due to intellectual property issues, you'll have to settle for whatever srpt is willing to reveal . . . moreover, it's not the FDA's responsibility to review/edit/police those communications . . . so investor beware . . . just sayin' . . .
. . . just the broad-brush explanation . . . thanks . . .
. . . Janet Woodcock may or may not have painted herself into a corner . . . but I do think her primary motivation was to make sure etep received a full and fair hearing and also to potentially soften the blow for parents going forward . . . I think a CRL is coming, but the terms will be relatively easy to fulfill if the drug actually works -- which I very much doubt . . . just sayin' . . .
. . . (excerpted from The New York Times article by Andrew Pollack, 4/25/16) . . . “The F.D.A. strongly urged Sarepta to do a larger study with a placebo control to better determine whether the drug worked. But the company, based in Cambridge, Mass., argued that it would be unethical and impractical to do so, since early hints of effectiveness meant that parents would no longer enroll their sons in a trial where they might not get the drug.
Instead, Sarepta compared the data from the 12 boys treated with eteplirsen to historical data from patients in Italy and Belgium who were as closely matched as possible in disease characteristics.” . . .
. . . save the right-wing drivel for your next trump rally . . . the FDA remains the global gold standard for government agencies charged with looking out for the health and safety of its citizens . . . period . . .
. . . don't get worked up, ci, I didn't say I thought the drug worked or that the company's platform was worthwhile -- I think they both stink big time . . . that said, I'm just here for the long-shot odds . . . took a flier years ago on Human Genome Sciences (HGSI) at $1.75 and sold the last of it near $30 . . . srpt, for me, is not an investment, it's just a recreational bet that I can afford to make . . . good luck . . .
. . . I'm not selling, c9, I'm just expressing the view that this pig remains a long shot -- which, as I've explained, is the only reason I'm holding it . . . I was simply lookin' for a little action . . . but no matter how anybody tries to spin it, this thing's got all the markings of a real stinker . . .
. . . very likely, the only thing that can save this stinker now is intervention by the anti-science, anti-government, right-wing congress . . . maybe they can lean on the FDA -- threaten to reduce their funding . . . or something equally stupid . . . in any case, there's still a chance this pig wins approval . . . (still holdin' my shares, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . (from The New York Times article by Andrew Pollack, 4/25/16) ""Dr. Billy Dunn, the director of the F.D.A.’s division of neurology products, opened the meeting by saying that the agency was “highly sensitive to the urgency” of finding drugs for muscular dystrophy. Still, he said, the F.D.A. was bound by law to approve drugs only if there was “substantial evidence” of effectiveness.. . . “These words are not vague words to be defined according to whim or fashion,” he said. “Anecdote and emotion do not change the data with which we are confronted, no matter the attendance,” he added."". . . spot on, Dr. Dunn . . . (still holdin' my shares, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . absolutely, a0, if the FDA determines the drug is worthless -- what's the point? . . . better to spend the money on dmd alternatives -- say from CATB, which has an oral drug that has shown real promise in early trials . . . these kids don't need another placebo . . . (and, yes, I own CATB, as well as this pig) . . .
. . . that's the expectation, folks -- great news, huh? . . . kids wouldn't have to endure a weekly infusion of any drug -- especially one that seems to offer only placebo-at-best performance . . . of course, a real cure would likely bury all pretenders in the dmd pharmacy space -- but who cares? . . . this is all about the kids . . . (still holdin' my srpt shares, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . I haven't been hiding, mi . . . you just haven't been paying attention -- maybe a habit you picked up in school . . . anyway, I don't currently own BMRN, but I do own shares of this pig -- like a lot of people, I like to bet on a real long shot now and then . . . good luck with your drisa research . . .
. . . "If this was looking like a CRL I believe he would be more public with either PRs on other parts of the business or behind the scenes rallying of the Moms.". . . you're joking right? . . .