is it obvious that the FDA was completely shiftless with everybody that it has been dealing with
they were ALL duped by the FDA ... with ppmd in the background (trust me on this ...)
and now the FDA is faced with this, after working so hard to "level the playing field" (hi pat furlong)
its a disgrace, and its been a disgrace for longer than investors knew
RNA should not survive that data ... but I bet it will, at least for now
I need a shower when I think about what appears to have gone on here
Simp, the important thing is totality of the data, a concept Ed Kaye kept emphasizing yesterday, based on prior experience with the FDA. The totality of SRPT data--duration of efficacy, production of dystrophin, clean safety profile--is better than RNA's across the board. I can't emphasize that enough. SRPT is superior in every measure. I doubt RNA can even be approved.
AA literally became a dirty word after months and months of everybody who was near the fda talking about how clearly srpt fit for AA
were they all that dumb, or were they misled?
I vote misled
publicly, temple sent signals that were ignored by all
and now we have rumors of rna P3 boys dropping out (I do not think these are just rumors)
will the final insult be that the fda requires srpt to do a full blown P3 because of what is happening with RNA?
will fda use the poor RNA d data to finally put a nail in the srpt AA coffin?
or will it just ask for another ton of data and go the back door way of dragging this out
the fda gave RNA BT status without seeing the full P2 results, KNOWING that those results absolutely had to be available but were not being released by GSK for reasons that could not be positive
could srpt have EVER gotten BT without full P2 results? I think not
great efforts were made to level the playing field, because after all, PF worked so hard to make sure the FDA thought about it that way
what a mess
"will the final insult be that the fda requires srpt to do a full blown P3 because of what is happening with RNA?"
Just because RNA's data stinks, and their AE's are even worse should not indicate that
SRPT's science is not orders of magnitude superior. You seem intent lately on linking SRPT
To Drisaspersen's poor results. Again....... I think that implication is faulty in a big way.
Drisa is based on inferior chemistry known to create off target negative side effects. Drisa is given in suboptimal dosages. Drisa did NOT show statistical significance in their primary endpoint - 6 mwt. To punish SRPT/Etep for the sins of a completely different drug would be the height of lunacy.
Etep works, and Drisa sucks, based on facts shared to date. It's not SRPT's fault that
their drug works and RNA's doesn't. In fact, the opposite should happen.......Drisa's poor results should launch Etep to the front once and for all. The short thesis on Etep was simply wrong.
Too bad. Shorts, MDA, Pat Furlong and others need to come to terms with the reality here.
Eteplirsen is going to own the Exon 51/DMD space whether they like it or not.
I wouldn't put anything past the FDA anymore. Their interests don't seem to be all that invested in the boys suffering from DMD, but rather bureaucracy and bait-and-switch tactics. Thankfully, I do think that a positive AdComm panel made up of doctors not working for the FDA should do the trick.
The FACTS for Pat Furlong, the FDA, and anyone else who can read, are that Etep creates statistically significant results in 6 mwt AND dystrophin production while exhibiting a pristine safety profile. Drisa does not show a similarly positive clinical impact while it does produce VERY significant adverse effects even at suboptimal dosing. If it stinks like a pig, squeals line a pig, and looks like a pig......... It's a PIG.
Etep is gonna own this space despite what MDA, Pat Furlong,and other GSK/RNA lovers claim.