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.
"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.