IMO Ruby has it right. The FDA sees GSK's drisapersen as a drug that may get to more patients
simply because it has the manufacturing ease and money to make it happen. This is good for the boys and will help to get them on antisense therapy to slow the disease progression. If drisapersen comes out first the boys can start with that and if they have trouble with it due to SAE's they can switch to eteplirsen down the road. CG has stated that in a previous conference call or article that we can be best in class and still win the race since this type of switching will delay disease progression and ultimately get them onto a safer drug while allowing the boys to wait as little as possible for treatment.
I think one of the unspoken fears (and some anger directed toward the FDA as a result of those fears) is that because now GSK has BTD for drisapersen then that somehow will translate to the FDA not giving us the AA wink we are expecting. I suspect this comes from the following thinking: "well the FDA did not abandon the dmd boys since they gave drisapersen BTD so they don't have to look like they are irrational and heartless with respect to helping the boys and the parents. i.e The FDA didn’t abandon them. And this, in turn, allows them to play tough with trial size and demand more patients in a phase 3 for followup. And that means we won't get the AA wink nor AA and will have to do a full phase 3 trial ." This, IMO, is irrational and ridiculous. IMO, the FDA would certainly like to have as many players as possible who can deliver life-saving drugs to the dmd boys for this terrible, and until antisense therapy, intractable disease. More drug options means more boys can be helped and allows some redundancy in case one drug has too many SAE's or is late due to manufacturing issues. In other words, giving GSK BTD is risk mitigation for the boys and not a negative for us.
Sentiment: Strong Buy
Fellow invetsors, this is the way I see it.
IMO, GSK did not ask the FDA to assess its chances to gain AA, because it knew it wouldn't have got it, given the side effects of its drug.
GSK would have had to show to the FDA its dystrophin data to ask for AA, but since it knew it had little chances to gain AA at present (because of the drug's side effects) it didn't give FDA the info on dystrophin, just because that would have helped indirectly SRPT to gain support for its own AA request, thereafter. So GSK went for BTD instead.
SRPT doesn't have the safety problems GSK has, so it can ask for AA, and to support it, it has to provide its dystrophin data. It is mainly about dystrophin now, in order to gain AA. GSK move not to give its dystrophin data was intended not to give support, indirectly, to SRPT request, hoping that SRPT data on dystrophin is not conclusive. But the benefit of the doubt, in this case, goes to accepting AA request, IMO, because the safety profile of Etiplirsen is good and because there are no other therapies available. Just IMHO.
Thanks jkadvd, I think you are right on the mark. I'm a little weak on intellectual property details, but I believe our key advantage centers on our morph-delivered chemistry. The door to an entire wonderland of exon skips is being cracked open, and we're first in line and best in class. Manufacturing ramp-up is what it is -- and even CG has referenced lotteries and other mechanisms that can determine who gets what when. I'm not sure if this is apropos, but it's almost as if the fda is hedging its bet, ensuring (like you cite) that more boys can obtain a drug that can address their regression. Call it a holding pattern -- and like our non-ambulent twins have proven, unless you get this elixir into the blood while there is still muscle mass to use it, you've missed the window to make a difference. And my recurring dream is that someone will share news that these twins are now able to record a score in the 6MWT. My, what a miracle that would be!
Sentiment: Strong Buy
Its not a zero sum game here--part 2....
The negative that people see is that we will have to split the market with GSK and thus a reduction in sales can occur. In fact, I think something different will happen. Instead, you create more confidence in the treatment market for dmd and can treat more patients worldwide thereby reducing risk when using this new antisense therapy and causing even more patients to be able to adopt it. The market can start larger than if just one solution is involved, and grow from there. More importantly, you validate the entire technology and platform more readily with more players. Sure, in a simplistic myopic sense it looks like less money for the short term but in reality these drugs are ushering in a new rational drug design paradigm. i.e. correct by construction --almost engineering approach-- as opposed to trying 1000's of different ideas and hoping one works. This is game changing technology and the more who adopt it, the better, for us investors and more importantly the patients.
Consider that with both drugs in place then cross licensing can occur allowing both drugs to be sold worldwide and let the patient/doctor decide. Given that our drug has lower SAE's I only see things getting better.
In other words, we can win, GSK can win, and most importantly patients can win. Its not GSK wins and we lose. And if anything its more like GSK loses given its inferior drug. If the FDA has any remote amount of common sense about a little david company like SRPT going up against a goliath like GSK then I would imagine they want both to be successful when treating these patients.
If for some reason, the FDA loses its mind and acts completely stupid and political and doesn't give us the AA wink then I will be more than happy to eat crow on this long winded diatribe I just wrote and promise to not subject anyone here to such a long post again. But I don't think crow will be on my plate--more like filet mignon and lobster. IMHO.
What happened, Piney? Your Ruby id not taken a seriously as you'd hoped ... and so you've got to drag your old pipe-smokin' "jkaudvid" puppet out of the your puppet trunk? (I see you slightly changed his monitker for some sly reason too!) .....Guess what? I haven't even read the post and I won't even bother.
I think you just wasted a lot of message board space putting out a narrative about underlying intentions of the FDA that only exist in your head. All this hand wringing over the manufacturing capacity is just ludicrous - eteplirsen will be meeting a market capacity of less than10,000 patients in the entire world and you people are talking about manufacturing as if it some great hurdle to overcome? BTD is simply the FDA recognizing that DMD is a life threatening disease that has no effective treatments and that based on GSK's early clinical data, they may have a drug that offers a new treatment paradigm. It says nothing about the drug's prospects to get to the market first, it says nothing about the ease or difficulty about manufacturing it, it says nothing about how deep the pockets are of the sponsor and, most importantly, it says nothing about the relative prospects or value of SRPT's drug - give it a rest!