When the FDA publicly states that it has no higher priority than DMD, it's logical to expect that it must "move the needle" to indicate that it has been doing SOMETHING to justify the delay. But what can the agency identify as the precise point at which it decided to take action that will bring etep to market? The drisa data are a candidate, but they almost force the FDA to admit how they never had the data properly in hand before the November guidance on etep. Input from the moms is important, but the FDA cannot admit that advocacy has any bearing on its decisions.
I think the tipping point will be a combination of our 6MWT data and a nod toward "established experts" who reinforced how 6MWD are the proper standards for objectively evaluating how patients respond to drugs aimed at DMD, a progressive disease. The FDA will cite how it's in-depth review of DMD "prompted a detailed examination" of 6MWT protocols -- which (remarkably) included agency representation at our 120-week tests. Bottom line is that the FDA can now cite etep's 6MWD as "objective evidence of clinical benefit." When coupled with confirmed production of dystrophin (i.e., our surrogate marker), the FDA can cite how it has concluded that it must take immediatel action to make this drug widely available to a population of boys who have no alternative therapy available to halt the progression of a deadly disease.
As my daughter would say: yada yada whatever. You were slow, FDA, but you eventually did the right thing. Now let's crank up the manufacturing conveyor belt and get moving on something we woulda/coulda/shoulda been doing months ago. And p.s.: please fast track our other exons. You DON'T want a repeat of this debacle, right?
When you consider what happened with ZGNX (and their dry Zohydro), AdCom voted 12-1 against Zohydro and FDA took almost 6 months longer than they initially indicated for CLR, eventually going against AdCom and approving the drug.
When FDA eventually gets this right (and I'm confident they will), they will come to a scientifically justified and validated conclusion that Eteplirsen provides a meaningful clinical benefit in the subset of exon 51 DMD children. It will just take them longer than the DMD community (which is far more important than us investors) would want.
The FDA has already said that the small sample size is not an issue, which somehow the bear camp chooses to ignore. Interestingly, the bear camp uses the recent comment by an FDA official (mis-attributed to SRPT), to bolster their position that FDA ALWAYS require a confirmatory ph3 trial (which is false).
The short part of the float will simply act as rocket fuel to push the share price up to at least $75 per share, on the initial positive news of AA. But there will be more, b/c Eteplirsen will pave the way for the next eons, which should have a similar safety/efficacy profile. And then there's the RNAi Marbug anti-viral platform. Altogether, SRPT's share price will be north of $200 w/i 2 years, and more after that.
I bought ZGNX (thanks Bionerd51) after the AdCom vote. It has been worth the wait ( I have a fx at C 7 and will ask for Zohydro in March). I have been waiting for SRPT since 09.2012. I think the wait will be worth it again!
This oft-repeated article of faith is baloney: "The FDA has already said that the small sample size is not an issue"......It's baloney because it's based on what some known FDA "softie" (Temple?) once said somewhere. As we now know what one guy in the FDA may say on one or more occassions really doesn't mean much, now does it? If 12 kids was all it took, then why would they want a phase 3 with more kids?
The 12 kid business is of course of SOME concern to SOMEONE but it's going to get dealt with by the phase 3 anyway. So it's SOMEONE's concern that is going to get dealt with IN SOME WAY. To make those someone's more or less happy.
So let's can the "a small trial not an issue with the FDA" fantasy/mantra....It IS an issue - but it's not a killer....it's a bump in the road - a speed bump, if you will.....And it's giving us enough time to get the needed amount of salable drug ready to be able to meet the market..... ..!....( Funny how that worked out, eh?)
You can ignore the big picture if you want to post day in and day out for a year or two about one program, sure......but the big picture is still looming over everything. And it's a pretty nice looking picture when seen from a safe distance, actually