After finally reading the full PR the FDA's request for additional safety and dystrophin as a surrogate marker from the current data set sends an unmistakable message. The FDA plans on making eterplirsen available to every exon 51 Duchennes patient in the U.S. Everyone has to remember that the FDA meeting was a post-PhaseII data sharing meeting so that Sarepta can receive guidance regarding the best path forward. Sarepta did not submit an entire NDA data package. FDA requesting additional data from this trial is very comforting for me as a longterm shareholder. Just recently, Isis was slapped-down by the FDA due to animal cancer data that they withheld from the FDA for their toxic " Chemical Christmas Tree" type drug, Kynamro. Dystrophin as a surrogate will help FDA members gain the confidence in approving eterplirsen. believe it or not their will be FDA members who don't know the complete role dystrophin plays in Muscular Dystrophy who will ultimately vote on this drug. Drug manufacturing will continue as per CG and NDA preparations and writing will begin in earnest. Something tells me that the FDA will designate Sarepta's entire DMD program as "Breakthrough Therapy" and will also include this program [all exons] for it's voucher program. Last comment...If we assume that the FDA is going to approve eterplirsen via AA or BT the realistic expected timeline for this will not be changed because of the FDA 's request. Sarepta will be challenged to submit an NDA by the end of 2013.
Recognizing change and adapting to it is crucial to successful investing. The US needs an industry in which it can lead the world forward. Traditional technology, computers and cell phones are no longer creating demand and driving productivity like they did a decade ago. We are on the cusp of a biological revolution.
I have to lower the bid this morning to make sure I get good execution. There's never been a better time to be overweight on this stock.