As announced by CG in November, the FDA decided it was "premature" for SRPT to file an NDA based on two things. A) the fact that Drisaspersen data showed ample dystrophin production without the expected positive clinical impact. And B) that the natural history, as shown in McDonalds study, and/or as understood by those in charge at FDA, could be used to explain the stability shown by the 12 kids in the Eteplirsen study.
Thing is, both of those pillars to the FDA argument were shown to be downright false. Period. The tactics that were used to get the FDA to the point where they believed A + B, were also shown to be questionable. Beyond
A + B being shown to be absolutely incorrect, we also had the FDA visit during the stellar 120 week 6 mwt testing. GSK abandoned RNA like Miley Cyrus gets rid of outfits. And.................the FDA got a preview of what ANY panel would recommend when 7 of the absolute brightest minds in the world all agreed that this drug works,and the kids need access to it NOW. Lastly, Bastings and his anti-SRPT no matter what agenda were sent away by JW.
Logic would dictate, that if the foundation for your reversal is shown to be absolutely false, while the data for Eteplirsen continues to literally shine, and the brightest DMD minds all agree the drug works with an astonishing lack of side effects, the FDA should then find themselves exactly where they were prior to being misled by A + B.........................waiting to receive the NDA from SRPT for Eteplirsen. Best of all, the data supporting Eteplirsen is now WAY better than it was when the FDA originally suggested that an NDA should be should be submitted. What argument against approval is left? BAstings just hates SRPT and doesn't care about proper protocol or data? Zero side effects MUST be a lie? The argument against AA seems to have crumbled since November, leaving only one valid choice - AA.
just for claritys sake. The FDA said previously they had NOT made any formal decision either way on AA, the market just interperted it that way. If I am wrong someone please correct me.
Sentiment: Strong Buy
I was thinking the same thing, but then I realized that literally EVERYTHING that the FDA reversal was based on has been shown to be wildly wrong, and on top of that, multiple other positive things have ensued since November. i just don't see how you justify not granting AA based on those things. Sure, I've got some puts to hedge my gains this time around, but the only decision that can be supported by facts, far as I can tell, is outright AA.
Well said Grey. New etep data plus suspect analysis by the FDA in November gives the Division of Neurology no reasonable place to hide. My hope is to give AA to all exon 51 deletion boys while agreeing to a study looking at non-ambulant boys. In my opinion it is this group that seems to get lost in the discussion and needs Etep as quickly as possible. Thus give it to them all!!!
The pulmonary etc. stabilization of the twins does seem to get overlooked, and I have not seen the nonambulatory population factored into revenue projections for etep. It should. The twins are the perfect poster children for the price associated with foot-dragging by the FDA. Every day, another young boy advances toward wheelchair confinement because etep is not available. And once in the wheelchair, they will continue to decline -- unless they gain access to etep to arrest the progression of DMD. I fail to understand how the FDA can possibly justify its intransigence and failure-to-act on a drug with NO AE'S !! They may believe they are protecting some people -- but NONE of those people have DMD.
Sentiment: Strong Buy