They are seeking full approval.Why start a confirmatory trial? And if they obtain full approval than the argument goes there is no unmet need. Better hope their approval is AA and not full.
I agree. What is your take on the Red Acre point about the risk of a possible BMRN full approval? I can't imagine anyone with a stake would want that other than BMRN. I expect the advocates to come out in force for dris but not to the extent it results in a full approval that limits choice and on such sketchy data.
You know over the last few years perhaps the only knock on your take on things is you have been a tad overly optimistic. Now put that in perspective as to how the cheerleaders rate. Hope you and the multiple personalities keep the cards and letters coming.
Next 60 days may be rough, but have to agree that getting dris approved is a benefit for etep approval. Any data release on etep during this period could really shake things up- either wk 216 walk data or new trial safety to debunk small N. The parents want options and what's wrong with that so long as there is choice.
Scientific advisory board. No conflict with Ed but a financial conflict with the organization which I believe has a financial stake in a BMRN approval.
Yes Starf. Now you have the real bear thesis. Influence across all key parts of the approval process. CG dug an awful deep hole there. At this point my hope is Etep gets swept up in the approval of the inferior drugs because those who can influence the process can get away with delay but not an outright rejection of Etep unless it fails by its own data along the way. That would draw too much attention.
Fired good post. There always are risks and they are priced in to some extent. Problems occur when idiots go blathering on about how there no risks and folks start drinking the koolaid. The best team on paper does not always win the game or even a series. Ignore most of the cheerleaders because they now have repeatedly established they have no idea what they are talking about or THEY lashout against charities- yes charities- and bureaucrats-what do you expect?- because THEY were wrong in their overly optimistic predictions. Personally I am not getting too caught up in the competition here and believe the FDA had reasons for no dual ADCOM, anything from preventing the circus it would have been to simply giving the company more time to offer safety data from the new trials and 216 week 6mw data. With all the noise around this process the one constant for the most part is that data has been our friend and that is a good thing.
That is right. There are people out there who make claims as fact when they really don't know what they are talking about. Those false prophets like those who made claims on the dystrophin results should come clean and not be the first to cast stones.
Well we will see. Prudent management practices may not permit them to delay--there would be no room for error and there remains no assurance this will sail through without some surprise or delay. If they role the dice and lose it will be a disaster of epic proportions.
You hope so but given the need for a secondary before ADCOM and the poor overall market conditions those holding the large short positions do not need to run to the exit because the exit will likely come to them. In other words I am not holding my breath for a short squeeze but will continue to ride the generally good profile of the drug. And risks do remain. Better to climb a wall of worry than rely on outlandish positive speculation that inflates expectations and misstates reality. BTW those folks are as harmful as those who spread misinformation for the dark side.
It is recent enough to still remember the pain of CG's bafoonery. I am OK with "recent" in that context. If that is the worst thing that influences the stock than I am OK with that too.
The nervousness may be a little overdone here. The fact that these experts will participate is a good thing and it offers the company the opportunity to explain what ithe data means before the haters get a shot at it (and you know they will out there-Bmrn). Remember the FDA had this data and filed and many good points made about disclosure requirements, cash raise etc. My point has been that this is no slam dunk and there can be wide variability in the data but remain cautiously optimistic.
Not mysterious--they need a panel. That may not be a bad thing but it should be enough to curb some of the rampant enthusiasm of the cheerleaders and which was the downfall of the CG regime in terms of over aggressive messaging that led to expectations which could not be met ( e.g. use of the term "stabilized") Also bios down generally
Why do you convene a panel of experts to confirm that when you look up the sky is blue? It just seems apparent the company wants the data to be viewed a certain way and it likely is not clear from the data itself--applause
If the data is not noisy why do you need a panel to explain it? I am not saying that I expect bad data but I think it is fair to constrain the cheerleader like enthusiasm. It appears to be that the data should be relatively good but it will require some expert commentary to explain why even if it is not so on its face.