I'm fairly certain that excitement was for SMA, Progeria, and Pompe's. He didn't explicitly state this, but mentioned being excited about "new targets." Those were the new targets mentioned a few months later, so it's a logical conclusion.
I agree with this. Although if drisa is approved, they will get some profit. It'll just evaporate fairly quickly if eteplirsen is also approved.
I think we can all agree these kids have been through enough, but given all of the questions that have dogged eteplirsen since P2, it is more than reasonable for an Adcom to review the drug, particularly with no FDA experts on staff. If you want to argue for a speedy approval once the Adcom has reviewed the drug, I'm right there with you. It should come swiftly and not wait until the PDUFA.
The FDA does not have any DMD experts on staff. Knowing this, do you really think they are going to skip an Adcom full of DMD experts. Really????????????????? Step away from the pipe. The Adcom is happening. If you don't believe me, ask the company what they think and ask the parents what they think. All you're doing is creating myth and lore. It does the company or the stock no good.
' I don't understand why folks here think it would be so unreasonable that the FDA might choose the latter course.'
Because the FDA has never at any time given any indication that such a thing would occur, and in fact, on multiple occasions have suggested their strict adherence to the review process. Why is this so hard to understand? No wonder so many shorts hate SRPT longs. It's because they refuse to use common sense.
And yes, it would be awesome if it happened. But it is not and has never been realistic.
Yeah, I doubt the Adcom date confirmation would create a move like this with this kind of volume. Would love to see Ed eff the shorts by releasing just enough of the data ahead of WMS to put them in their place.
Gary posts useful information at times, so I don't care to completely ignore him. But I'm also not going to sit idle while he creates hype that is more likely to do harm than good.
That's because you frequently neglect to use common sense, gary. Did you read the email from the Jett Foundation this morning? They themselves said there would be an Adcom. (As did PPMD and other DMD groups.) So you need to remove "parents" from your statement. Maybe replace it with the word "ignorance".
I'm hoping for a beej from a pretty analyst. Doubt it happens though since I won't be there.
That's true, but Hereditary orotic aciduria is an extreme case in that it has about 20 patients worldwide. How many experts do you think there are to treat the condition? Enough to fill an Adcom panel with 16 members? Doubtful. You bring up a good point, but given that, by the FDA's own admission, they are not experts on DMD, does it really make sense that they won't have a panel of experts weigh in?
THE ADCOM ISN'T GOING TO BE BYPASSED! Why are you completely ignoring this company's history with the FDA? You can't pick and choose to focus on one good meeting all the while the FDA has continuously spread doubt. When are we going to get out of dreamland?
1. There is absolutely no precedent for the FDA to skip an Adcom for a new drug to treat a previously untreatable disease. None. The FDA hasn't exactly done anything unprecedented thus far with eteplirsen. Why are they going to start now?
2. The FDA has repeatedly stated that there will be an Adcom to review DMD drugs.
3. PPMD just had a seminar on Adcoms. Um, because there will be an Adcom.
Common sense, people. Let's be realistic. We want an Adcom. The likelihood of approval goes way up with what is almost guaranteeably going to be a very positive recommendation.
I'm always amazed at how well you can peg people from behind a computer screen! You really like riding the delusional train, don't you?